A new study shows that subretinal adeno-associated virus 8 (AAV8) injections cause proinflammatory T-cell immune response against the transgene product. According to the study, published in the ...
An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent cells and leading to syndromic hearing loss. In a new experimental study, ...
Treatment with fidanacogene elaparvovec, a recombinant adeno-associated virus (AAV) vector developed for the treatment of hemophilia B, led to sustained expression of the high-activity factor IX ...
Pharmaceutical Technology on MSN
AviadoBio broadens vMiX gene silencing licence with King’s College London
The expanded licensing lets AviadoBio pursue partnerships for broader applications, including cardiovascular, nephrology, ...
AI-assisted adeno-associated virus (AAV) capsid engineering has transformative potential in terms of accelerating and optimizing the development of gene therapies. That’s the view of Fangzhi Tan, PhD, ...
The efficacy of adeno-associated virus (AAV)-mediated gene therapy for the inner ear is fundamentally constrained by the natural tropism of viral vectors, which often lack the precision and efficiency ...
Researchers from Amsterdam UMC have overturned a key assumption in the biological pacemaker field. In a new preclinical study ...
Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic ...
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