Hemophilia. Cystic fibrosis. Duchenne muscular dystrophy. Huntingtons disease. These are just a few of the thousands of disorders caused by mutations in the bodys DNA. Treating the root causes of these debilitating diseases has become possible only recently, thanks to the development of genome editing tools such as CRISPR, which can change DNA sequences in cells and tissues to correct fundamental errors at the sourcebut significant hurdles must be overcome before genome-editing treatments are ready for use in humans.
Enter the National Institutes of Health Common Funds Somatic Cell Genome Editing (SCGE) program, established in 2018 to help researchers develop and assess accurate, safe and effective genome editing therapies for use in the cells and tissues of the body (aka somatic cells) that are affected by each of these diseases.
Todaywith three ongoing grants totaling more than $6 million in research fundingDuke University is tied with Yale University, UC Berkeley and UC Davis for the most projects supported by the NIH SCGE Program.
In the 2019 SCGE awards cycle, Charles Gersbach, the Rooney Family Associate Professor of Biomedical Engineering, and collaborators across Duke and North Carolina State University received two grants: the first will allow them to study how CRISPR genome editing affects engineered human muscle tissues, while the second project will develop new CRISPR tools to turn genes on and off rather than permanently alter the targeted DNA sequence. This work builds on a 2018 SCGE grant, led by Aravind Asokan, professor and director of gene therapy in the Department of Surgery, which focuses on using adeno-associated viruses to deliver gene editing tools to neuromuscular tissue.
There is an amazing team of engineers, scientists and clinicians at Duke and the broader Research Triangle coalescing around the challenges of studying and manipulating the human genome to treat diseasefrom delivery to modeling to building new tools, said Gersbach, who with his colleagues recently launched the Duke Center for Advanced Genomic Technologies (CAGT), a collaboration of the Pratt School of Engineering, Trinity College of Arts and Sciences, and School of Medicine. Were very excited to be at the center of those efforts and greatly appreciate the support of the NIH SCGE Program to realize this vision.
For their first grant, Gersbach will collaborate with fellow Duke biomedical engineering faculty Nenad Bursac and George Truskey to monitor how genome editing affects engineered human muscle tissue. Through their new project, the team will use human pluripotent stem cells to make human muscle tissues in the lab, specifically skeletal and cardiac muscle, which are often affected by genetic diseases. These systems will then serve as a more accurate model for monitoring the health of human tissues, on-target and off-target genome modifications, tissue regeneration, and possible immune responses during CRISPR-mediated genome editing.
Currently, most genetic testing occurs using animal models, but those dont always accurately replicate the human response to therapy, says Truskey, the Goodson Professor of Biomedical Engineering.
Bursac adds, We have a long history of engineering human cardiac and skeletal muscle tissues with the right cell types and physiology to model the response to gene editing systems like CRISPR. With these platforms, we hope to help predict how muscle will respond in a human trial.
Gersbach will work with Tim Reddy, a Duke associate professor of biostatistics and bioinformatics, and Rodolphe Barrangou, the Todd R. Klaenhammer Distinguished Professor in Probiotics Research at North Carolina State University, on the second grant. According to Gersbach, this has the potential to extend the impact of genome editing technologies to a greater diversity of diseases, as many common diseases, such as neurodegenerative and autoimmune conditions, result from too much or too little of certain genes rather than a single genetic mutation. This work builds on previous collaborations between Gersbach, Barrangou and Reddy developing both new CRISPR systems for gene regulation and to regulate the epigenome rather than permanently delete DNA sequences.
Aravind Asokan leads Dukes initial SCGE grant, which explores the the evolution of next generation of adeno-associated viruses (AAVs), which have emerged as a safe and effective system to deliver gene therapies to targeted cells, especially those involved in neuromuscular diseases like spinal muscular atrophy, Duchenne muscular dystrophy and other myopathies. However, delivery of genome editing tools to the stem cells of neuromuscular tissue is particularly challenging. This collaboration between Asokan and Gersbach builds on their previous work in using AAV and CRISPR to treat animal models of DMD.
We aim to correct mutations not just in the mature muscle cells, but also in the muscle stem cells that regenerate skeletal muscle tissue, explainsAsokan. This approach is critical to ensuring long-term stability of genome editing in muscle and ultimately we hope to establish a paradigm where our cross-cutting viral evolution approach can enable efficient editing in multiple organ systems.
Click through to learn more about the Duke Center for Advanced Genomic Technologies.
Continue reading here:
Duke Researchers Garner Over $6 Million in NIH Funding to Fight Genetic Diseases - Duke Today
- Viewpoint: Anti-GMO arguments seem silly after 28 years of false narratives about health harms and and genetic ... - Genetic Literacy Project - March 29th, 2024 [March 29th, 2024]
- Viewpoint: Here's how genetically engineered fruits and vegetables will soon emerge as a grocery store 'selling point ... - Genetic Literacy Project - March 29th, 2024 [March 29th, 2024]
- CRISPR Investments: What We Heard at World Agri-Tech - Seed World - March 29th, 2024 [March 29th, 2024]
- When SpaceX's Starship is ready to settle Mars, will we be? (op-ed) - Space.com - September 17th, 2023 [September 17th, 2023]
- Genome-wide promoter responses to CRISPR perturbations of ... - Nature.com - September 17th, 2023 [September 17th, 2023]
- Expanding the toolbox for RNA editing | ASU News - ASU News Now - September 17th, 2023 [September 17th, 2023]
- Electrical Engineer Named MIT Technology Review Innovator Under ... - University of California San Diego - September 17th, 2023 [September 17th, 2023]
- Can we rely on our 'moral force-field' to stop cloning going too far? - The National - September 17th, 2023 [September 17th, 2023]
- Otsuka Collaborates with ShapeTX for Development of AAV Gene ... - Pharmaceutical Technology Magazine - September 17th, 2023 [September 17th, 2023]
- Cancer discovery earns U of A grad the Breakthrough Prize - University of Alberta - September 17th, 2023 [September 17th, 2023]
- The Brave New World of synthetic humans | Gne Taylor - IAI - September 17th, 2023 [September 17th, 2023]
- Targeting Tumors with Photosynthetic Bacteria - Optics & Photonics News - September 17th, 2023 [September 17th, 2023]
- Artificial Photosynthesis Breakthrough Researchers Produce ... - SciTechDaily - September 17th, 2023 [September 17th, 2023]
- BASF patent on watermelons upheld: European Patent Office rejects ... - Bio Eco Actual - September 17th, 2023 [September 17th, 2023]
- Space Industry Is Growing Faster Than Its Workforce, Analysts Say - Slashdot - September 17th, 2023 [September 17th, 2023]
- New 'Inverse Vaccine' Shows Potential to Treat MS and Other ... - Slashdot - September 17th, 2023 [September 17th, 2023]
- Gene therapy: Donor DNA may protect babies from certain disorders - Medical News Today - May 18th, 2023 [May 18th, 2023]
- Viewpoint: Grim consequences of Greenpeace's war on ... - Genetic Literacy Project - May 18th, 2023 [May 18th, 2023]
- Predicting Diabetic Kidney Disease with the Use of a Novel Algorithm - Genetic Engineering & Biotechnology News - May 18th, 2023 [May 18th, 2023]
- Quantum biology on horizon? How futuristic physics theory could ... - Study Finds - May 18th, 2023 [May 18th, 2023]
- Scientists can collect human DNA from water, air, and basically ... - Earth.com - May 18th, 2023 [May 18th, 2023]
- The First Crispr-Edited Salad Is Here - WIRED - May 18th, 2023 [May 18th, 2023]
- Gene editing technology used to produce disease-resistant calf - Earth.com - May 18th, 2023 [May 18th, 2023]
- Neanderthals passed down their tall noses to modern humans ... - Livescience.com - May 18th, 2023 [May 18th, 2023]
- Squid Camouflage Inspires Human Invisibility: Is it Possible? - DISCOVER Magazine - May 18th, 2023 [May 18th, 2023]
- Mice Model Technologies Market Poised for 7.4% CAGR Growth, Reaching USD 3.2 Bn by 2031 | Transparency Market Research - Yahoo Finance - May 18th, 2023 [May 18th, 2023]
- Google Cloud launches A.I.-powered tools to accelerate drug discovery, precision medicine - CNBC - May 18th, 2023 [May 18th, 2023]
- How Fauci, scientists with ties to Wuhan lab persuaded the ... - U.S. Right to Know - May 18th, 2023 [May 18th, 2023]
- The Daunting Task of Cutting Heavy Metals from Baby Food - Undark Magazine - May 18th, 2023 [May 18th, 2023]
- Ingestible Device Profiles and Peers into the Microbiome and ... - Genetic Engineering & Biotechnology News - May 18th, 2023 [May 18th, 2023]
- X-Men Officially Names the Best Powers Any Mutant Can Have - Screen Rant - May 18th, 2023 [May 18th, 2023]
- 10 Forgotten Marvel Comics Characters That Debuted In The '80s - Screen Rant - May 18th, 2023 [May 18th, 2023]
- 18 Human Genetic Engineering - Clemson University - April 19th, 2023 [April 19th, 2023]
- Pros and Cons of Genetic Engineering - Benefits and Risks - March 31st, 2023 [March 31st, 2023]
- What is Genetic Engineering? Types, Process & Applications - March 31st, 2023 [March 31st, 2023]
- How artificial skin is made and its uses, from treating burns to skin cancer - South China Morning Post - March 31st, 2023 [March 31st, 2023]
- Welcome to the UNC Department of Genetics | Department of Genetics - February 5th, 2023 [February 5th, 2023]
- Global Genetically Modified Crops Market Is Projected To Grow At A 6% Rate Through The Forecast Period - EIN News - February 5th, 2023 [February 5th, 2023]
- Science and History of GMOs and Other Food Modification Processes - February 2nd, 2023 [February 2nd, 2023]
- Genetics | History, Biology, Timeline, & Facts | Britannica - January 31st, 2023 [January 31st, 2023]
- Human genetics | Description, Chromosomes, & Inheritance - January 31st, 2023 [January 31st, 2023]
- Genetic testing - Mayo Clinic - January 31st, 2023 [January 31st, 2023]
- Genetic Disorders: What Are They, Types, Symptoms & Causes - January 31st, 2023 [January 31st, 2023]
- Colossal Biosciences Project to Revive the Prehistoric Woolly Mammoth Raises Staggering $60 Million Series A Funding - Nature World News - January 22nd, 2023 [January 22nd, 2023]
- 2023 is going to witness a surge in technology courses top courses that will remain in vogue - Times of India - January 22nd, 2023 [January 22nd, 2023]
- M.R.S. Rao birthday: All you need to know about the Padma Shri winning Indian scientist - Free Press Journal - January 22nd, 2023 [January 22nd, 2023]
- Russian cosmism - Wikipedia - January 8th, 2023 [January 8th, 2023]
- Postgenderism - Wikipedia - January 8th, 2023 [January 8th, 2023]
- LessWrong - Wikipedia - January 8th, 2023 [January 8th, 2023]
- Eliezer Yudkowsky - Wikipedia - January 8th, 2023 [January 8th, 2023]
- Genetic Engineering Principles of Biology - December 26th, 2022 [December 26th, 2022]
- Engineering the Perfect Baby | MIT Technology Review - December 26th, 2022 [December 26th, 2022]
- Genetic Engineering Science Projects - Science Buddies - December 21st, 2022 [December 21st, 2022]
- DSI adoption at COP15 can financially help protect biodiversity in India: Experts - The Tribune India - December 21st, 2022 [December 21st, 2022]
- Tel Aviv University researchers demonstrate success of potential one-time vaccine to treat HIV/AIDS - ETHealthWorld - December 21st, 2022 [December 21st, 2022]
- Should You Buy 22nd Century Group Inc (XXII) Stock After it Has Risen 14.29% in a Week? - InvestorsObserver - October 28th, 2022 [October 28th, 2022]
- Home :: National Institute for Biotechnology and Genetic Engineering - October 11th, 2022 [October 11th, 2022]
- A recently discovered law of physics could help predict genetic mutations - October 11th, 2022 [October 11th, 2022]
- Farmers, consumers will embrace GMOs if they understand them - The Standard - October 11th, 2022 [October 11th, 2022]
- Synthetic Biology Market is Expected to Report a CAGR of ~21% from 2021 to 2029: Industry Size, Growth & Forecast at Douglas Insights - Yahoo... - October 11th, 2022 [October 11th, 2022]
- After 45 years of science-based art, Esther Klein Gallery winds down - WHYY - October 11th, 2022 [October 11th, 2022]
- Behind this Nobel prize is a very human story: theres a bit of Neanderthal in all of us - The Guardian - October 11th, 2022 [October 11th, 2022]
- The key to tastier beer might be mutant yeastwith notes of banana - Popular Science - October 11th, 2022 [October 11th, 2022]
- Why You'll Probably Never See Cotton Candy Grape Wine - Tasting Table - October 11th, 2022 [October 11th, 2022]
- Earth materials in technology The National - The National - October 11th, 2022 [October 11th, 2022]
- CULINARY THRILL SEEKING Proceed with caution. It's hotter than blazes season. - Port Arthur News - The Port Arthur News - October 11th, 2022 [October 11th, 2022]
- Give legal rights to animals, trees and rivers, say experts - The Guardian - October 11th, 2022 [October 11th, 2022]
- How yeast DNA may help protect astronauts from cosmic radiation in space - EastMojo - October 11th, 2022 [October 11th, 2022]
- Life finds a way, but should it? The ethics of genetic engineering - The Trinitonian - October 8th, 2022 [October 8th, 2022]
- To modify or not to modify? Genetic Modification and Gene Editing - A divergence by the UK - Lexology - October 8th, 2022 [October 8th, 2022]
- Gene Therapy Hits Its Stride in the Clinic - Genetic Engineering & Biotechnology News - October 8th, 2022 [October 8th, 2022]
- GWAS, MWAS and mGWAS provide insights into precision agriculture based on genotype-dependent microbial effects in foxtail millet - Nature.com - October 8th, 2022 [October 8th, 2022]
- Making Sense of the Latest Apple-iPhone News - The Motley Fool - October 8th, 2022 [October 8th, 2022]
- Gene therapy brings hope to people with sickle cell, HIV - Monitor - October 8th, 2022 [October 8th, 2022]
- GMO: Everything you need to know about lifting ban - FarmKenya Initiative - The Standard - October 8th, 2022 [October 8th, 2022]
- Century Therapeutics to Present at the SITC 37th Annual Meeting - Yahoo Finance - October 8th, 2022 [October 8th, 2022]
- Cultured meat could help solve the climate crisis. Heres what it will take to move it from the lab to the dinner table - Fortune - October 8th, 2022 [October 8th, 2022]
- Poseida Therapeutics to Present Trial in Progress Poster for Phase 1 P-MUC1C-ALLO1 Study at the Society for Immunotherapy of Cancer Annual Meeting -... - October 8th, 2022 [October 8th, 2022]
- Explained: How scientists engineered mosquitoes that will cut the transmission of malaria - Firstpost - October 8th, 2022 [October 8th, 2022]
- Here Are the Biggest Health Industry News Items of 2022 So Far - DocWire News - October 8th, 2022 [October 8th, 2022]