Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...
Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic defect.
Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
GlobalData on MSN
Genespire to take rare disease gene therapy to clinic in 2026
GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.
Stocktwits on MSN
Rare stock slips 3% today — what’s up with Ultragenyx’s gene therapy for Sanfilippo syndrome?
Ultragenyx now expects a 6-month review period for the therapy and a likely decision from the FDA regarding potential ...
The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...
Gene therapy drugs have the potential to cure some diseases, but some have a price tag of over a million dollars. Who gets access to them and who doesn't?
Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...
MedPage Today on MSN
FDA Halts Two Gene Therapy Trials After Child Develops Brain Tumor
Studies of Hunter and Hurler syndromes on hold ...
The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...
(Yicai) Jan. 28 -- Chinese medical researchers have made a breakthrough in gene therapy that overcomes the limitations of ...
University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...
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