IN the summer of 2019, a mother in Nashville, Tennessee in the United States, with a seemingly incurable genetic disorder finally found an end to her suffering by editing her genome.
Victoria Grays recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research gene therapy.
I have hoped for a cure since I was about 11, the 34-year-old said.
Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.
Over several weeks, Grays blood was drawn so that doctors could get to the cause of her illness stem cells from her bone marrow that were making deformed red blood cells.
The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 pronounced Crisper a new tool informally known as a molecular scissors.
The genetically-edited cells were transfused back into Grays veins and bone marrow. A month later, she was producing normal blood cells.
Medics warn that caution is necessary, but theoretically, she has been cured.
This is one patient. This is early results. We need to see how it works out in other patients, said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.
But these results are really exciting.
In Germany, a 19-year-old woman was treated with a similar method for a different blood disease beta thalassemia.
She had previously needed 16 blood transfusions per year. Nine months later, she is completely free of that burden.
For decades, the DNA of living organisms such as corn and salmon has been modified. But Crispr, invented in 2012, made gene editing more widely accessible.
It is much simpler than preceding technology, cheaper and easy to use in small labs.
The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.
Its all developing very quickly, said French geneticist Emmanuelle Charpentier, one of Crisprs inventors and the co-founder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.
Crispr was the latest breakthrough in a year of great strides in gene therapy, a medical adventure that started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.
Scientists practising the technique insert a normal gene into cells containing a defective gene.
It does the work the original could not, such as making normal red blood cells in Grays case or making tumour-killing super white blood cells for a cancer patient.
Crispr goes even further: instead of adding a gene, the tool edits the genome itself.
After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.
They join several other gene therapies bringing the total to eight approved in recent years to treat certain cancers and an inherited blindness.
Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.
Twenty-five, 30 years, thats the time it had to take, he said. It took a generation for gene therapy to become a reality. Now, its only going to go faster.
Just outside Washington, at the US National Institutes of Health (NIH), researchers are also celebrating a breakthrough period.
We have hit an inflection point, said US NIHs associate director for science policy Carrie Wolinetz.
These therapies are exorbitantly expensive, however, costing up to US$2 million (RM8.18 million) meaning patients face grueling negotiations with their insurance companies.
They also involve a complex regimen of procedures that are only available in wealthy countries.
Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion and fighting a general infection.
You cannot do this in a community hospital close to home, said her doctor.
However, the number of approved gene therapies will increase to about 40 by 2022, according to Massachusetts Institute of Technology (MIT) researchers.
They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.
In this Oct 10, 2018, photo, He speaks during an interview at his laboratory in Shenzhen, China. The scientist was recently sentenced to three years in prison for practicing medicine illegally and fined 3 million yuan (RM1.76 million). AP
Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who dont necessarily share the medical ethics of Western medicine.
In 2018 in China, scientist He Jiankui triggered an international scandal and his excommunication from the scientific community when he used Crispr to create what he called the first gene-edited humans.
The biophysicist said he had altered the DNA (deoxyribonucleic acid) of human embryos that became twin girls Lulu and Nana.
His goal was to create a mutation that would prevent the girls from contracting HIV (human immunodeficiency virus), even though there was no specific reason to put them through the process.
That technology is not safe, said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr scissors often cut next to the targeted gene, causing unexpected mutations.
Its very easy to do if you dont care about the consequences, he added.
Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.
The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.
There is also the temptation to genetically edit entire animal species, e.g. malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.
The researchers in charge of those projects are advancing carefully however, fully aware of the unpredictability of chain reactions on the ecosystem.
Charpentier doesnt believe in the more dystopian scenarios predicted for gene therapy, including American biohackers injecting themselves with Crispr technology bought online.
Not everyone is a biologist or scientist, she said.
And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies crops?
Charpentier thinks that technology generally tends to be used for the better.
Im a bacteriologist -- weve been talking about bioterrorism for years, she said. Nothing has ever happened. AFP Relaxnews
We're sorry, this article is unavailable at the moment. If you wish to read this article, kindly contact our Customer Service team at 1-300-88-7827. Thank you for your patience - we're bringing you a new and improved experience soon!
Article type: metered
User Type: anonymous web
Campaign ID: 7
Cxense type: free
User access status: 3
- human genetics | Description, Chromosomes, & Inheritance ... - May 19th, 2020
- Human genetics - Wikipedia - May 19th, 2020
- COVID-19 Pandemic: Global Risks of More Complex Character and the Visions of the Future World - Valdai Discussion Club - May 19th, 2020
- Rothamsted turn to harvesting coronavirus data - Lab News - May 19th, 2020
- Complement genes add to sex-based vulnerability in lupus and schizophrenia - UAB News - May 19th, 2020
- DU to set up School of Public Health - The Indian Express - May 19th, 2020
- Covid-19 research: 45 Bengaluru startups working on medicine, testing methods and vaccine - Economic Times - May 19th, 2020
- Immortalized Cell Line Market Development, Trends, Key Driven Factors, Segmentation And Forecast to 2020-2026 - Cole of Duty - May 19th, 2020
- Worlds Shortest Population Reveal the Largest Genetic Contributor to Height - Technology Networks - May 15th, 2020
- Group of Genes Have Altered Expression in Autism - Technology Networks - May 15th, 2020
- Why can two young and healthy individuals be affected so differently by coronavirus? - Health24 - May 15th, 2020
- Amgen To Present At The Bank Of America Merrill Lynch Virtual Global Healthcare Conference - BioSpace - May 15th, 2020
- Viewpoint: Darwin's 'Descent of Man' is both deeply disturbing and more relevant than ever - Genetic Literacy Project - May 15th, 2020
- Study Finds Low Proportion of Individuals With Autism Receive Recommended Genetic Tests - Technology Networks - May 15th, 2020
- Cats can catch Covid-19 from other cats. The question is: Can we? - STAT - May 15th, 2020
- Deficient Expression of DGCR8 in Human Testis is Related to Spermatoge | IJGM - Dove Medical Press - May 15th, 2020
- Prevail Therapeutics Reports First Quarter 2020 Financial Results and Business Highlights - GlobeNewswire - May 15th, 2020
- Fulcrum Therapeutics, Inc. (FULC) Q1 2020 Earnings Call Transcript - The Motley Fool - May 15th, 2020
- Scientists concerned that coronavirus is adapting to humans - The Guardian - May 11th, 2020
- Coronavirus quickly spread around the world starting late last year, new genetic analysis shows - CNN - May 11th, 2020
- Your genes could determine whether the coronavirus puts you in the hospital and we're starting to unravel which ones matter - The Conversation US - May 11th, 2020
- Yes, COVID-19 is mutating, here's what you need to know - ABC News - May 11th, 2020
- Conservatives Are Not the Only Ones Who Ignore Facts and the Science - Merion West - May 11th, 2020
- Coronavirus in Scotland: Charity warns Covid will cause a spike in ME cases - as it calls for 'harmful' exercise treatment to be banned -... - May 11th, 2020
- Dr. Misaki Wayengera: The Man Behind Uganda's Covid 19 Test Kits - New Vision - May 11th, 2020
- Its In The Genes? Scientists Think Coronavirus Exploits Silent Hidden Mutations In The Body - International Business Times - May 11th, 2020
- From blood clots to 'Covid toe': Experts confounded by series of medical mysteries - The Straits Times - May 11th, 2020
- MET 2020 Slot booking to commence on July 15, Examination dates available at manipal.edu - Jagran Josh - May 11th, 2020
- Vitagene Launches The First FDA Authorized Saliva based Zero Contact COVID-19 At Home Test - Business Wire - May 10th, 2020
- Genetics in focus after coronavirus deaths of siblings and twins - The Guardian - May 10th, 2020
- 'An anvil on my chest': What it's like to have COVID-19 - LancasterOnline - May 10th, 2020
- Coronavirus may have spread to humans as early as October 2019 - study - The Jerusalem Post - May 10th, 2020
- Team reveals genomic history of ancient civilizations in the Andes - UC Santa Cruz - May 10th, 2020
- Regeneron Reports First Quarter 2020 Financial and Operating Results - BioSpace - May 10th, 2020
- Scientists at Ben-Gurion University of the Negev Develop Anti-Coronavirus Surface Coating Based on Nanomate... - The Auto Channel - May 10th, 2020
- COVID-19 and food security - Fitzwilliam College, University of Cambridge - May 10th, 2020
- Val Sheffield elected to the American Academy of Arts and Sciences - Iowa Now - May 1st, 2020
- DNA gives clues into risk of developing Alzheimer's disease and other dementias - Alabama NewsCenter - May 1st, 2020
- Rare Gene Discovered That Nearly Doubles Risk of Developing a Neurodegenerative Disease - Clinical OMICs News - May 1st, 2020
- Progress in understanding the genetic basis of mental health - SFARI News - May 1st, 2020
- Coronavirus was widespread in UK at very start of pandemic, says genetics expert - Sky News - May 1st, 2020
- This is how you do the genetics heritage filter on Instagram that everyone's doing - The Tab - May 1st, 2020
- COVID-19 vaccine in Ireland could take a year and a half - IrishCentral - May 1st, 2020
- MRC scientists elected Fellows of the Royal Society - Cambridge Network - May 1st, 2020
- Parkinson's discovery implicates "second brain" in the gut - New Atlas - May 1st, 2020
- Humans: are we the most effective vector of disease? - BugBitten - BMC Blogs Network - May 1st, 2020
- Could genetics explain why some COVID-19 patients fare worse than others? - Live Science - April 27th, 2020
- Human Genetics Market Overview, Top Companies, Region, Application and Global Forecast by 2026 - Latest Herald - April 27th, 2020
- American Academy of Arts & Sciences Elects UVM's Wallace to Its Membership - UVM News - April 27th, 2020
- The PBS documentary The Gene showcases genetics promise and pitfalls - Science News - April 9th, 2020
- Few clinical trials are done in Africa: COVID-19 shows why this urgently needs to change - The Conversation Africa - April 9th, 2020
- UCLA web app will enlist publics help in slowing the spread of COVID-19 - Newswise - April 9th, 2020
- Why does the new coronavirus kill some people and barely affect others? - Wilkes-Barre Citizens Voice - April 9th, 2020
- COVID-19: Few Clinical Trials are Done in Africa. This Needs to Change ASAP. - The Wire - April 9th, 2020
- 'Behavioral suppression' needed to decrease coronavirus infections in Japan: experts - The Mainichi - April 9th, 2020
- The secret call of the wild: how animals teach each other to survive - The Guardian - April 9th, 2020
- Yann Joly on the fight against genetic discrimination - McGill Reporter - April 2nd, 2020
- Science to the rescue? How modern genetics could help save the world from coronavirus - Genetic Literacy Project - April 2nd, 2020
- Oldest human genetic data gleaned from 1.8-million-year-old tooth Haaretz - News Collective - April 2nd, 2020
- Science to the rescue? How modern genetics could help save the world from coronavirus - Alliance for Science - Alliance for Science - April 2nd, 2020
- BHU department claims to have discovered new technology to test COVID-19 - Jagran Josh - April 2nd, 2020
- Stealth BioTherapeutics Reports Fiscal Year 2019 Financial Results And Recent Business Highlights - BioSpace - April 2nd, 2020
- Researchers at U of T developing antibodies to 'neutralize' novel coronavirus before it invades cells - News@UofT - April 2nd, 2020
- What is coronavirus and Covid-19? An explainer - KTVZ - April 2nd, 2020
- Plasmid Market was Valued at US$ 89.52 million in 2018 and is Estimated to Reach US$ 447.68 Million by 2027, growing at a CAGR of 19.5% over the... - April 2nd, 2020
- The genetic architecture of the human cerebral cortex - Science Magazine - March 26th, 2020
- The Coronavirus Pandemic Shows Us The Importance Of Combatting Climate Change - Forbes - March 26th, 2020
- Kallyope Inc. Announces $112M Series C Financing to Support First Clinical Trials and Advance Portfolio of Programs Targeting the Gut-Brain Axis - P&T... - March 26th, 2020
- IN CONSERVATION: DR. JEFF STROVEL, CEO of VERALOX THERAPEUTICS - BioBuzz - March 26th, 2020
- How healthtech startup Bione aims to use genetic testing in the fight against coronavirus - YourStory - March 26th, 2020
- Coronavirus: Massive gap in US response revealed after scientists learn colleague tested positive through twee - MEAWW - March 26th, 2020
- Avera announces ability to test for COVID-19 in South Dakota - The Dickinson Press - March 24th, 2020
- University of Utah experts advise caution over drugs hyped as possible coronavirus treatments - Salt Lake Tribune - March 24th, 2020
- Invitae and Muscular Dystrophy Association (MDA) Expand Access to No-Charge Genetic Testing in the US and Canada - PRNewswire - March 24th, 2020
- Studying the African genome could yield new medical treatments for everyone - Genetic Literacy Project - March 24th, 2020
- Human Genetics Market Higher Growth Rate / CAGR over the Forecast Period to 2026 by Key Players like QIAGEN, Agilent Technologies, Illumina - New Day... - March 16th, 2020
- Coronavirus is hard on older people and scientists aren't sure why - NBCNews.com - March 16th, 2020
- Researchers study irregular horse heartbeats, hoping to find a cure - Minnesota Daily - March 16th, 2020
- Dr. John T. Macdonald Foundation seeking to fund community-based organizations with major grant dollars - Miami's Community Newspapers - March 16th, 2020
- A book that could save lives: Adam Rutherford's How to Argue with a Racist reviewed - Spectator.co.uk - March 16th, 2020