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Category Archives: Gene Therapy

Bioinformatics approach helps researchers find new use for old drug

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Developing and testing a new anti-cancer drug can cost billions of dollars and take many years of research. Finding an effective anti-cancer medication from the pool of drugs already approved for the treatment of other medical conditions could cut a considerable amount of time and money from the process.

Now, using a novel bioinformatics approach, a team led by investigators at Beth Israel Deaconess Medical Center (BIDMC) has found that the approved antimicrobial drug pentamidine may help in the treatment of patients with advanced kidney cancer. Described online in the journal Molecular Cancer Therapeutics, the discovery reveals how linking cancer gene expression patterns with drug activity might help advance cancer care.

"The strategy of repurposing drugs that are currently being used for other indications is of significant interest to the medical community as well as the pharmaceutical and biotech industries," says senior author Towia Libermann, PhD, Director of the Genomics, Proteomics, Bioinformatics and Systems Biology Center at BIDMC and Associate Professor of Medicine at Harvard Medical School. "Our results demonstrate that bioinformatics approaches involving the analysis and matching of cancer and drug gene signatures can indeed help us identify new candidate cancer therapeutics."

Renal cell cancer consists of multiple subtypes that are likely caused by different genetic mutations. Over the years, Libermann has been working to identify new disease markers and therapeutic targets through gene expression signatures of renal cell cancer that distinguish these different cancer subtypes from each other, as well as from healthy individuals. In this new paper, he and his colleagues were looking for drugs that might be effective against clear cell renal cancer, the most common and highly malignant subtype of kidney cancer. Although patients with early stage disease can often be successfully treated through surgery, up to 30 percent of patients with renal cell cancer present with advanced stages of disease at the time of their diagnosis.

To pursue this search, they made use of the Connectivity Map (C-MAP) database (http://www.broadinstitute.org/cmap), a collection of gene expression data from human cancer cells treated with hundreds of small molecule drugs.

"C-MAP uses pattern-matching algorithms to enable investigators to make connections between drugs, genes and diseases through common, but inverse, changes in gene expression," says Libermann. "It provided us with an exciting opportunity to use our renal cell cancer gene signatures and a new bioinformatics strategy to match kidney cancer gene expression profiles from individual patients with gene expression changes inducted by various commonly used drugs."

After identifying drugs that may reverse the gene expression changes associated with renal cell cancer, the investigators used assays to measure the effect of the selected drugs on cells. This led to the identification of a small number of FDA-approved drugs that induced cell death in multiple kidney cancer cell lines. The investigators then tested three of these drugs in an animal model of renal cell cancer and demonstrated that the antimicrobial agent pentamidine (primarily used for the treatment of pneumonia) reduced tumor growth and enhanced survival. Gene expression experiments using microarrays also identified the genes in renal cell cancer that were counteracted by pentamidine.

"One of the main challenges in treating cancer is the identification of the right drug for the right individual," explains first author Luiz Fernando Zerbini, PhD, of the International Center for Genetic Engineering and Biotechnology in Cape Town, South Africa, adding that this bioinformatics approach could be a particularly valuable lower-cost model in developing countries.

The authors say their next step will be to evaluate the potential of pentamidine in combination with the current standard-of-care therapies to treat kidney cancer. "Since the drugs we are evaluating are already FDA-approved, successful studies in preclinical animal models may enable us to rapidly move these drugs into clinical trials," adds Libermann.

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Bioinformatics approach helps researchers find new use for old drug

Gene Therapy Used to Preserve Sight in Patients

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Newswise Orlando, Fla. In two separate studies, vision scientists have developed healthy genes to prevent blinding diseases that stem from genetic defects. The research is being presented at the 2014 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) this week in Orlando, Fla.

In a clinical trial to treat choroideremia, a rare disease that causes progressive and irreversible blindness, scientists developed a virus that can replace the missing gene (that causes the disease) in the cells at the back of the eye. Six months after the virus was injected into patients, findings showed that some patients experienced improved vision.

Abstract Title: Improved visual function in patients with choroideremia undergoing subretinal gene therapy Presentation Start/End Time: Sunday, May 4, 3:15 3:30pm Location: S 320AB Session Number: 147

In a separate study, researchers developed a gene therapy to stop the progression of a form of retinitis pigmentosa, an inherited disease transmitted from mothers to sons. Two years after the therapy was used to treat dogs at an early stage of the disease, the treatment remained effective. Further use of the technique in dogs with mid and late stages of the disease also resulted in a positive response to the intervention.

Abstract Title: RPGR gene augmentation delivered at early, mid and late stage disease in a canine model of XLRP rescues photoreceptor structure and function Presentation Start/End Time: Tuesday, May 6, 11am 12:45pm Location: Exhibit/Poster Hall SA Session Number: 342 # # #

The Association for Research in Vision and Ophthalmology (ARVO) is the largest eye and vision research organization in the world. Members include some 11,500 eye and vision researchers from over 70 countries. ARVO encourages and assists research, training, publication and knowledge-sharing in vision and ophthalmology.

All abstracts accepted for presentation at the ARVO Annual Meeting represent previously unpublished data and conclusions. This research may be proprietary or may have been submitted for journal publication.

Embargo policy: Journalists must seek approval from the presenter(s) before reporting data from paper or poster presentations. Press releases or stories on information presented at the ARVO Annual Meeting may not be released or published until the conclusion of the presentation.

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Gene Therapy Used to Preserve Sight in Patients

Regeneron, Avalanche To Develop Novel Gene Therapy Products In Ophthalmology

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By RTT News, May 05, 2014, 08:34:00 AM EDT

(RTTNews.com) - Regeneron Pharmaceuticals, Inc. ( REGN ) and Avalanche Biotechnologies, Inc. formed a broad collaboration to discover, develop and commercialize novel gene therapy products to treat ophthalmologic diseases. The collaboration includes novel gene therapy vectors and proprietary molecules, discovered jointly by Avalanche and Regeneron, and developed using the Avalanche Ocular BioFactory, an adeno-associated virus (AAV)-based, proprietary, next-generation platform for the discovery and development of gene therapy vectors for ophthalmology.

As part of the agreement, Avalanche would receive an upfront cash payment, contingent payments of up to $640 million upon achievement of certain development and regulatory milestones, plus a royalty on worldwide net sales of collaboration products. The collaboration comprises up to eight distinct therapeutic targets, and Regeneron will have exclusive worldwide rights for each product it moves forward in clinical development. Further, Avalanche has the option to share in development costs and profits for products directed toward two collaboration therapeutic targets selected by Avalanche.

Pursuant to the agreement, Regeneron has a time-limited right of first negotiation for certain rights to AVA-101, Avalanche's gene therapy product targeting vascular endothelial growth factor currently under development for the treatment of wet age-related macular degeneration, upon completion of the ongoing Phase 2a study.

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Regeneron, Avalanche To Develop Novel Gene Therapy Products In Ophthalmology

Gene discovery links cancer cell 'recycling' system to potential new therapy

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1-May-2014

Contact: Leslie Orr Leslie_Orr@urmc.rochester.edu University of Rochester Medical Center

University of Rochester scientists have discovered a gene with a critical link to pancreatic cancer, and further investigation in mice shows that by blocking the gene's most important function, researchers can slow the disease and extend survival.

Published online by Cell Reports, the finding offers a potential new route to intrude on a cancer that usually strikes quickly, has been stubbornly resistant to targeted therapies, and has a low survival rate. Most recent improvements in the treatment of pancreatic cancer, in fact, are the result of using different combinations of older chemotherapy drugs. The research led by Hartmut "Hucky" Land, Ph.D., and Aram F. Hezel, M.D., of UR Medicine's James P. Wilmot Cancer Center, identifies a new target in the process of garbage recycling that occurs within the cancer cell called autophagy, which is critical to pancreatic cancer progression and growth.

Autophagy is derived from the Greek roots "auto" (self) and "phagein" (to eat), and is an intracellular digestive process that allows cells to survive under stress. During a cell's transformation from normal to malignant, autophagy speeds up to keep pace with rapid cellular changes and a tumor's quest to grow. The newly discovered PLAC-8 gene sustains the highly active recycling process, as it removes faulty proteins and organelles and degrades them into reusable building blocks during cancer progression.

"What makes this an exciting opportunity is that the gene we're studying is critical to the cancer cell's machinery but it is not essential to the function of normal cells," said Land, chair of Biomedical Genetics at the University of Rochester School of Medicine and Dentistry and director of research at Wilmot. "By targeting these types of non-mutated genes that are highly specific to cancer, we are looking for more effective ways to intervene."

The Cell Reports study underlines Wilmot's overall unique approach to cancer research. Rather than investigate single faulty genes linked to single subtypes of cancer, Rochester scientists have identified a larger network of approximately 100 non-mutated genes that cooperate and control the shared activities of many cancers. While investigating this larger gene network, Land and Hezel focused on PLAC-8.

Moreover, the team found that by inactivating PLAC-8 in mice and shutting down autophagy, they could significantly slow cancer's progression. The relevance of PLAC-8 may also extend to other tumors lung, colon, and liver, for example -- that share key genetic changes such as KRAS and p53 mutations that are present in the majority of pancreatic cancers. The breadth of these findings is an area of ongoing study in the Land and Hezel labs.

"PLAC-8 and its job within the cancer cell of accelerating recycling suggests new points of attack and what we all hope will be opportunities to identify and develop new treatments," said Hezel, vice chief of Wilmot's Division of Hematology and Oncology and a UR associate professor. "Our data showing PLAC 8's role in autophagy has great potential because while there are other drugs being evaluated to inhibit autophagy, not all of them target proteins specifically important to this process in tumors."

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Gene discovery links cancer cell 'recycling' system to potential new therapy

Novel Financing For Gene Therapy Company

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By Cathy Yarbrough, Contributing Editor

Gene therapy pioneer Katherine High, M.D., was looking forward to her first meeting in 2011 with Jeffrey Marrazzo, then a consultant to the CEO of Childrens Hospital of Philadelphia (CHOP). A veteran of three life sciences companies, Marrazzo was meeting with Dr. High and other CHOP leaders to identify potential new revenue streams for the hospital.

Dr. High, an international leader in gene therapy research and clinical application, had considered postponing the meeting because she was so busy with her work as director of the hospitals Center for Cellular and Molecular Therapeutics (CCMT). However, she did not reschedule because she wanted to ask Marrazzo for a favor: Could he speak with the VCs who were calling her and inquiring about investing in CCMTs work on RPE65?

I hadnt spoken to them yet, because at the time I was busier than usual with my patient care, research, and teaching responsibilities. In addition, VCs are not a constituency that I normally deal with, said Dr. High, professor of pediatrics at the University of Pennsylvania as well as a Howard Hughes medical investigator.

Scheduled to last just 60 minutes, Dr. Highs first meeting with Marrazzo stretched to seven hours and was followed by many more meetings to determine the best approach for advancing CCMTs gene therapy discoveries. The result was a commitment of $50 million from CHOP to fund a new biotech company, Spark Therapeutics, to design, evaluate, and commercialize gene therapies for disorders that can lead to blindness, hemophilia, and neurodegenerative diseases. The company, like the hospital, is headquartered in Philadelphia.

CHOPs serving as the sole equity investor in Spark is definitely a novel financing model for early corporate activities to develop novel therapeutics, said Marrazzo, now president, CEO, and cofounder of Spark. Every situation is unique, and the situation should dictate the model.

Sparks situation was unusual because long before the companys official launch in late 2013, many assets were already in place, said Marrazzo, who uncovered them during his seven-hour conversation with Dr. High. It was like peeling back the layers of an onion, with each layer representing another asset, he said.

The assets included two clinical trials, a Phase 3 trial to treat a rare form of hereditary blindness, and a Phase 1/2 trial targeting hemophilia B, as well as staff members with gene therapy expertise in regulatory affairs, clinical research, and the manufacture of clinical grade vectors to transport genetic material into targeted cells.

Assembled at the center were world experts in gene therapy, said Marrazzo. CHOP had been incubating a biotech company within its four walls.

GENE THERAPY ASSETS UNDERVALUED Before investing $50 million to launch and operate Spark Therapeutics, CHOP officials considered but ruled out a licensing deal with an existing biopharm company or a start-up with VC funding. While we did have licensing deals on the table, that route would not have recognized the value of the asset in part because of the broad retrenchment that had occurred in the industry after the tragic 1999 death of Jesse Gelsinger in a gene therapy clinical trial, said Dr. High. Gelsinger died while participating in a clinical trial conducted by a University of Pennsylvania lab not connected to CCMT or CHOP.

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Novel Financing For Gene Therapy Company

Gene therapy may help hearing

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Australian researchers are trying a novel way to boost the power of cochlear implants: They used the technology to beam gene therapy into the ears of deaf animals and found it improved hearing.

The approach isn't ready for human testing, but it's part of growing research into ways to let users of cochlear implants experience richer, more normal sound.

Normally, microscopic hair cells in a part of the inner ear called the cochlea detect vibrations and convert them to electrical impulses that the brain recognizes as sound. Hearing loss typically occurs as those hair cells are lost, whether from aging, exposure to loud noises or other factors.

Cochlear implants substitute for the missing hair cells, sending electrical impulses to directly activate auditory nerves in the brain. They've been implanted in more than 300,000 people. While highly successful, they don't restore hearing to normal, missing out on musical tone, for instance.

The idea behind the project: Perhaps a closer connection between the implant and the auditory nerves would improve hearing. Those nerves' bushlike endings can regrow if exposed to nerve-nourishing proteins called neurotrophins. Usually, the hair cells would provide those.

Researchers at Australia's University of New South Wales figured out a new way to deliver one of those growth factors.

They injected a growth factor-producing gene into the ears of deafened guinea pigs, animals commonly used as a model for human hearing. Then they adapted an electrode from a cochlear implant to beam in a few stronger-than-normal electrical pulses.

That made the membranes of nearby cells temporarily permeable, so the gene could slip inside. Those cells began producing the growth factor, which in turn stimulated regrowth of the nerve.

-- from wire reports

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Gene therapy may help hearing

Cochlear implant enhances patient experience through gene therapy

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According to the World Health Organization, more than 360 million people worldwide live with disabling hearing loss, and for many, devices such as hearing aids and cochlear implants allow them to maintain a normal life style. But what if a cochlear device could offer a biological solution that would enhance a patients experience?

For the first time, researchers at the University of New South Wales in Australia have used cochlear implants to regenerate auditory nerves through gene therapy, a process where therapeutic DNA is inserted into cells to treat a disease.

Cochlear implants work by converting sounds into electrical signals that are sent directly to the auditory nerve, bypassing the outer and middle ear. The process allows for significantly improved hearing, including the ability to maintain a phone conversation, but the sounds they provide for patients are monotone and robotic.

Ultimately, we hope that after further research, people who depend on cochlear implant devices will be able to enjoy a broader dynamic and tonal range of sound, which is particularly important for our sense of the auditory world around us and for music appreciation, says Professor Gary Housley, Director of the Translational Neuroscience Facility at UNSW Medicine.

In 1993 multiple labs discovered that mammals ears would have the ability to regenerate cells if triggered according to the National Organization for Hearing Research Foundation, but until now there hadnt been a safe or efficient way to deliver the necessary proteins to the cochlear area.

We think its possible that in the future this gene delivery would only add a few minutes to the implant procedure, says the papers first author, Jeremy Pinyon, whose PhD is based on this work. The surgeon who installs the device would inject the DNA solution into the cochlea and then fire electrical impulses to trigger the DNA transfer once the implant is inserted.

Gene therapy research has provided hope for a number of genetic disorders and diseases, including cancer, HIV and multiple sclerosis.

"Our work has implications far beyond hearing disorders, says co-author Associate Professor Matthias Klugmann, from the UNSW Translational Neuroscience Facility research team. Gene therapy has been suggested as a treatment concept even for devastating neurological conditions and our technology provides a novel platform for safe and efficient gene transfer into tissues as delicate as the brain.

The research was recently published in the journal Science Translational Medicine.

Professor Housley discusses the new gene delivery technique in the UNSW video below.

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Cochlear implant enhances patient experience through gene therapy

mice-science-AFPrelax-250414.jpg

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April 25, 2014

Alzheimer's, caused by toxic proteins that destroy brain cells, is the most common form of dementia. AFP/Relaxnews pic, April 25, 2014.Spanish scientists have for the first time used gene therapy to reverse memory loss in mice with Alzheimer's, an advance that could lead to new drugs to treat the disease, they said Wednesday.

The Autonomous University of Barcelona team injected a gene which causes the production of a protein that is blocked in patients with Alzheimer's into the hippocampus a region of the brian essential to memory processing in mice that were in the initial stages of the disease.

"The protein that was reinstated by the gene therapy triggers the signals needed to activate the genes involved in long-term memory consolidation," the university said in a statement.

Gene therapy involves transplanting genes into a patient's cells to correct an otherwise incurable disease caused by a failure of one or another gene.

The finding was published in The Journal of Neuroscience and it follows four years of research.

"The hope is that this study could lead to the development of pharmaceutical drugs that can activate these genes in humans and allow for the recovery of memory," the head of the research team, Carlos Saura, told AFP.

Alzheimer's, caused by toxic proteins that destroy brain cells, is the most common form of dementia.

Worldwide, 35.6 million people suffer from the fatal degenerative disease, which is currently incurable, and there are 7.7 million new cases every year, according to a 2012 report from the World Health Organisation.

In 2010 the total global societal cost of dementia was estimated to be US$604 billion, according to Alzheimer's Disease International, a federation of Alzheimer associations around the world. AFP/Relaxnews, April 25, 2014.

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Gene Therapy May Enhance Cochlear Implants, Animal Study Finds

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By Steven Reinberg HealthDay Reporter

WEDNESDAY, April 23, 2014 (HealthDay News) -- Australian researchers say that gene therapy may one day improve the hearing of people with cochlear implants, allowing them to appreciate music and hear in noisy environments.

In experiments with deaf guinea pigs, senior study author Gary Housley and colleagues found that inserting genes in the area of the cochlear implant and passing an electric charge through the implant stimulated the growth of cochlear cells.

"Our study found a [new] way to provide safe localized delivery of a gene to the cochlea, using the cochlear implant device itself. The gene acts as a nerve growth factor, which stimulates repair of the cochlear nerve," said Housley, a professor and director of the Translational Neuroscience Facility at the University of New South Wales, in Sydney.

The cochlear implant is surgically placed in the cochlea, in the inner ear. The implant works by using a line of small electrodes within the cochlea to selectively stimulate cochlear nerve fibers at different positions and enhancing different sounds, or frequencies, Housley explained.

"In the cochlea of a person with good hearing, sound vibrations are encoded by sensory cells, called 'hair cells,' which stimulate the cochlear nerve fibers," he said. "With hearing loss, the hair cells are lost, and without them the cochlear nerve fibers die and retract into the bone within the core of the cochlea."

This makes the job of the cochlear implant difficult as the amount of electrical current needed to stimulate the nerves is quite high, Housley added.

The gene therapy, which makes the cells close to the electrode produce the nerve growth factor, causes the nerve fibers to grow out to those cells -- and therefore to the electrodes, he explained. This means that much less current is needed, so more selective groups of nerve fibers can be stimulated.

"In the future, people with cochlear implants may get this gene therapy at the time of their implant, and the computer system -- which is part of the cochlea implant that converts sound to electrical pulses along the array of electrodes -- should be able to provide a better sound perception," Housley said.

Scientists note, however, that research with animals often fails to provide similar results in humans.

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Gene Therapy May Enhance Cochlear Implants, Animal Study Finds

Cochlear Implant Also Uses Gene Therapy to Improve Hearing

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The electrodes in a cochlear implant can be used to direct gene therapy and regrow neurons.

Growth factor: The cochlear nerve regenerates after gene therapy (top) versus the untreated cochlea from the same animal (bottom).

Researchers have demonstrated a new way to restore lost hearing: with a cochlear implant that helps the auditory nerve regenerate by delivering gene therapy.

The researchers behind the work are investigating whether electrode-triggered gene therapy could improve other machine-body connectionsfor example, the deep-brain stimulation probes that are used to treat Parkinsons disease, or retinal prosthetics.

More than 300,000 people worldwide have cochlear implants. The devices are implanted in patients who are profoundly deaf, having lost most or all of the ears hair cells, which detect sound waves through mechanical vibrations, and convert those vibrations into electrical signals that are picked up by neurons in the auditory nerve and passed along to the brain. Cochlear implants use up to 22 platinum electrodes to stimulate the auditory nerve; the devices make a tremendous difference for people but they restore only a fraction of normal hearing.

Cochlear implants are very effective for picking up speech, but they struggle to reproduce pitch, spectral range, and dynamics, says Gary Housley, a neuroscientist at the University of New South Wales in Sydney, Australia, who led development of the new implant.

Cyborg cavy: An Xray image shows the cochlear implant in the left ear of a guinea pig.

When the ears hair cells degrade and die, the associated neurons also degrade and shrink back into the cochlea. So theres a physical gap between these atrophied neurons and the electrodes in the cochlear implant. Improving the interface between nerves and electrodes should make it possible to use weaker electrical stimulation, opening up the possibility of stimulating multiple parts of the auditory nerve at once, using more electrodes, and improving the overall quality of sound.

Peptides called neurotrophins can encourage regeneration of the neurons in the auditory nerve. Housley used a common process, called electroporation, to cause pores to open up in cells, allowing DNA to get inside. It usually requires high voltages, and it hasnt found much clinical use, but Housley wanted to see whether the small, distributed electrodes of the cochlear implant could be used to achieve the effect.

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Cochlear Implant Also Uses Gene Therapy to Improve Hearing

Gene therapy shows promise to help people regrow auditory nerve cells

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A new study out of Australia has promising potential for patients across the globe who use cochlear implants. Photo by Flickr user ryanjpoole

A new study outlines how gene therapy could reverse hearing loss and deafness. This may be music to the ears of the roughly 300,000 patients across the globe that depend on cochlear implants to hear.

Australian researchers published their findings Wednesday in the journal Science Translational Medicine. By stimulating gene cells, which were injected into the ear canal with electrical impulses, chemically deafened guinea pigs were able to regrow auditory nerve cells.

The scientists used guinea pigs as test subjects because of the similarities between the ear canals of humans and guinea pigs. While the researchers noted just how effective cochlear implants have been to date in helping those with profound hearing loss, they also noted their limitations. They hope to overcome those limitations through their research.

People with cochlear implants do well with understanding speech, but their perception of pitch can be poor, so they often miss out on the joy of music, said the studys senior author Gary Housley, a professor of neuroscience at the University of South Wales.

The cochlea is a tiny seashell-shaped organ located in the inner ear. It is filled with groups of microscopic hair cells that move in response to vibrations, and then convert those vibrations into electrical impulses that are carried to the brain and interpreted as sound. In some peoples ears, either because of genetics, old age, poisoning or loud noises, those tiny hair cells are damaged or lost and scientists havent found a way found to regrow them yet. In certain patients who experience profound hearing loss, a cochlear implant with electrodes can help stimulate whatever nerve cells are left.

With this study, Housley and his colleagues encouraged the production of neurotrophins, small proteins that stimulate the growth and maintenance of the hair-like nerve cells. They injected small rings of DNA, called plasmids, into the inner ear of the guinea pigs. Then, they exposed the animals cochleas to electrical currents that mimicked the electrical impulses provided to human cochleas through cochlear implants. By doing so, the membranes of the guinea pigs cells became more permeable to the injected DNA. The result triggered the production of neurotrophins and thus, the regrowth of nerve cells. The researchers are hoping that, in human subjects, they can achieve similar results.

While the researchers were ecstatic over the results, some of their enthusiasm was tempered because in some guinea pigs, results began to taper after three to six weeks. They hope to continue studying the application of gene therapy going forward.

The development of electrode array gene delivery may not only improve the hearing of cochlear implant recipients but also find broader therapeutic applications, Housely said. [Gene therapy] could be used to treat a range of neurological disorders, from Parkinsons disease to psychiatric disorders.

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Gene therapy shows promise to help people regrow auditory nerve cells

Hear me now? Gene therapy improves 'bionic ear' technology

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A procedure that uses a series of electric jolts to inject lab-designed DNA molecules into cells of the inner ear may help to regrow auditory nerves in people with profound hearing loss, according to researchers.

In a paper published Wednesday in Science Translational Medicine, Australian researchers said they used tiny electrodes and gene therapy to regenerate nerve cells in chemically deafened guinea pigs.

The procedure, they said, may one day improve the functioning of human cochlear implants -- electronic devices that provide hearing sensations to the deaf.

"People with chochlear implants do well with understanding speech, but their perception of pitch can be poor, so they often miss out on the joy of music," said senior author Gary Housley, a professor of neuroscience at the University of South Wales.

"Ultimately we hope that after further research, people who depend on cochlear implant devices will be able to enjoy a broader dynamic and tonal range of sound," Housely said in a prepared statement.

Houseley and his colleagues studied the procedure on guinea pigs because the structure of their inner ear is similar to that of humans.

The cochlea is shaped like a snail's shell, and is filled with a multitude of tiny hair cells that move in response to sound vibrations. Those vibrations are then converted into electrical nerve impulses that are carried to the brain.

If the hair cells are lost or damaged due to age, genetics, chemical poisoning or loud noise, they will not grow back. In some people who are profoundly deaf, an electrode may be implanted within the cochlea that can stimulate some nerve cells.

While cochlear implants help roughly 300,000 patients throughout the world to detect and interpret speech, researchers believe they can be improved if nerve cells are encouraged to grow closer to the electrode. In this latest study, Housely and his colleagues set out to stimulate growth in spiral ganglion neurons in guinea pigs.

Study authors believed they could do this by causing inner ear cells to produce neurotrophins, proteins that control the development, maintenance and function of nerve cells.

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Hear me now? Gene therapy improves 'bionic ear' technology


  1. Gene Therapy