Hereditary spastic paraplegia: Journey to a treatment - Boston Children's Answers
Researchers and parents collaborate to find a possible solution for a rare form of hereditary spastic paraplegia.| Boston Children's Answers
Researchers and parents collaborate to find a possible solution for a rare form of hereditary spastic paraplegia.| Boston Children's Answers
After expatriation of a partnership agreement with Astellas, Taysha Gene Therapies regained full rights to TSHA-102, its experimental gene therapy being developed to treat Rett syndrome. “We are thrilled to regain full global rights to our lead TSHA-102 Rett syndrome program, which enables full strategic flexibility and optionality as we continue to advance the program […] The post Taysha regains full rights to TSHA-102 gene therapy for Rett appeared first on Rett Syndrome News.| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
One-time treatment with the gene therapy FLT201 (avigbagene parvec) may help control Gaucher disease without the need for additional therapies, according to new data from four patients in an early clinical trial. Spur Therapeutics, the company developing FLT201, presented the data at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress, held […] The post One-time gene therapy controls Gaucher disease symptoms appeared first on Gaucher Disease News.| Gaucher Disease News
Shape Therapeutics’ SHP-201, an experimental gene therapy for Parkinson’s disease, significantly reduced the levels of alpha-synuclein — a protein that plays a key role in the development of the neurodegenerative […] The post Parkinson’s gene therapy reduced alpha-synuclein in animal models appeared first on Parkinson's News Today.| Parkinson's News Today – The Web's Daily Resource for Parkinson's News
Treatment introduces an expansion of the scope of gene therapies that might lead to similar treatments for neurodegenerative diseases in other parts of the body The post FDA approves novel gene therapy delivery platform for a rare retinal disorder first appeared on Science-Based Medicine.| Science-Based Medicine
When I received my wife’s recent text, which she wrote in all caps — something she’s morally opposed to — the room seemed to tilt. I felt like dancing like […] The post Is gene therapy for Huntington’s the miracle we’ve been waiting for? appeared first on Huntington's Disease News.| Huntington's Disease News – The Web's Daily Resource for Huntington's Disea...
Gene therapies can take place either 'in vivo' or 'ex vivo'; we compare these two approaches and their importance in gene therapy| Genomics Education Programme
Ever wonder how medicine and science converge to create new opportunities and new hope for the tiniest patients around the world? Cassidy Callahan, Rady Children’s Hospital-San Diego’s clinical research navigator, can certainly fill you in. Working in step with investigators and physician-scientists across Rady Children’s research team and partner organizations, Cassidy is a central part…| Rady Children's Health
What if your body already had everything it needed to heal, but no one told you how to use it? Dr. Adeel Khan, Founder of Eterna Health, has been cracking the code on regenerative medicine—helping people heal, recover, and even slow down aging using stem cells, peptides, and gene therapy.| The Mindvalley Podcast With Vishen Lakhiani | Listen Now
Cedars-Sinai researchers boost ACE enzyme in brain immune cells, dramatically reducing Alzheimer's plaques and reversing cognitive decline in mice.| Drug Discovery and Development
Mass Eye and Ear researcher Zheng-Yi Chen, DPhil, is studying several approaches to reverse different forms of inherited deafness that he hopes can one day pave the way for treatments for many genetic and non-genetic forms of hearing loss. Since the introduction of the cochlear implant more than 60 years ago, effective therapies for children… Read More »Opening the Gates to Gene Therapy for Treating Deafness The post Opening the Gates to Gene Therapy for Treating Deafness appeared first o...| FOCUS
Researchers Zheng-Yi Chen, DPhil, and Qin Liu, MD, PhD, are combining forces to combat a rare disease that leads to blindness and deafness. Watch this video of Drs. Chen and Liu describing their research at the 2022 Sense-ation! Gala, and hear from patient Hannah Corderman on how an Usher syndrome diagnosis has impacted her life. Usher syndrome… Read More »Racing the Clock and Raising Hope for Usher Syndrome Patients The post Racing the Clock and Raising Hope for Usher Syndrome Patients...| FOCUS
A Food and Drug Administration official who stepped down less than two weeks ago is getting his job back as the agency’s top vaccine regulator. Dr.| AP News
CRISPR-based gene editing technology Casgevy has been approved for treatment of sickle cell disease and beta-thalassaemia in the NHS| Genomics Education Programme
Neurogene received clearance from the FDA for a pivotal trial of NGN-401, which could support an application for the gene therapy's approval.| Rett Syndrome News
The first porcine kidney transplant (in a live recipient) It’s been a year, and like many, I have been waiting for the details to be published in the NEJM (where else – especially since this was done at MGH). Here are the key elements that caught my eye but if you are truly interested, read […]| Recon Strategy
The FDA has cleared CAP-003, a gene therapy for Parkinson’s associated with GBA mutations, to be tested in clinical trials.| Parkinson's News Today
New AAV proviral plasmid for vector manufacturing has potential to increase patient safety and make gene therapies more affordable, researchers say.| Pediatrics Nationwide
The Humans of the Wyss (HOW) series features members of the Wyss community discussing their work, the influences that shape them as professionals, and their collaborations at the Wyss Institute and beyond. Sayo Eweje loves finding solutions, whether he’s looking at a Rubik’s Cube or a technical challenge in the lab. When faced with the problem...| Wyss Institute
By Lindsay Brownell (BOSTON) — Getting medicines into the cells they’re designed to treat is a perennial problem for the medicine and pharmaceutical industries, and patients often suffer from side effects and ineffective treatments as a result. Current drug delivery vehicles carry a host of risks and limits: lipid nanoparticles can cause immune reactions and...| Wyss Institute
US neurologists are optimistic about the potential for a new Huntington's disease treatment - preferably a gene therapy - per a market survey.| Huntington's Disease News
The first four Rett syndrome patients treated with the gene therapy candidate NGN-401 have seen meaningful gains in skills, data show.| Rett Syndrome News
NGN-401 is one of six picked, three each by the Center for Biologics Evaluation and Research, and the Center for Drug Evaluation and Research.| Rett Syndrome News
Findings from researchers in Canada and Maryland support further development of gene therapy for treating people with fragile X syndrome.| Fragile X News Today
Meet the microscopic technique that turns big DNA strands into decipherable code for treating immune disorders.| Scienceline
A gene therapy to increase nerve cell connectivity worked to ease motor dysfunction in an ALS mouse model but did not prolong survival.| ALS News Today
The US regulatory agency acts to support the potential gene therapy's development, with ongoing Phase 1/2 trial showing safety in patients.| Rett Syndrome News
On June 17, 2024, the Department of Health and Human Services Office of Inspector General (OIG) issued an advisory opinion, approving a plan to provide| Health Industry Washington Watch
Let us take a look at how the hunt for amyotrophic lateral sclerosis (ALS) drugs has advanced over the past few years.| Labiotech.eu
Explore 10 gene therapy companies operating within the biotech industry, as each one hopes to develop their own novel candidates.| Labiotech.eu
Using the safe and sensible CRISPR technology that has the power to help the 300,000 babies born with sickle cell disease each year and the nearly 10 million people estimated to suffer from this genetic disorder globally.| science.thewire.in
