As gene therapy evolves to target additional indications—including more common indications—the choice of delivery platform is bound to become more interesting. At present, the most common delivery ...
In a recent review published in Signal Transduction and Targeted Therapy, researchers presented recombinant adeno-associated virus (rAAV)-based genetic applications to treat human diseases. Study: ...
There are numerous severe, difficult-to-treat monogenetic diseases that are caused by a defect in a single gene—also in the liver. These include the blood coagulation disorders hemophilia A or B or ...
What Are AAV Vector-Based Therapies? Gene therapies are groundbreaking approaches utilizing viral and non-viral vectors. These vectors introduce therapeutic genes into patient cells to treat diseases, ...
HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy ...
Adeno-associated viral vectors comprise the majority of recent gene therapy development programs due to their broad tissue-tropism and relatively low immunogenicity. Recent gene therapy approvals, ...
WATERTOWN, Mass.--(BUSINESS WIRE)--Dyno Therapeutics, Inc., a genetic technologies company applying artificial intelligence (AI) to solve the grand challenge of in vivo gene delivery, announced ...
SGT-003 gene therapy showed significant microdystrophin expression and muscle integrity improvements in DMD patients, with no serious adverse events reported. The novel AAV vector demonstrated ...
Non-human primate study results demonstrate improvements in skeletal and cardiac muscle delivery efficacy, safety potential, human translatability, and manufacturability over prior AAV capsids ...
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