The gene therapy regimen seems to be working wonders, including restoring nearly all hearing to a seven-year-old patient ...

While gene therapy clinical trials for peripheral artery disease ( PAD) using plasmid DNA or adenoviral vectors show no clear benefit in amputation-free survival, amputation, and all-cause mortality, ...

Bioluminescent imaging is helping researchers better understand the effectiveness of tissue regeneration enhancers delivered to injured heart tissue by different adeno-associated virus vectors.

JCR Pharmaceuticals has signed a licence agreement with Alexion, AstraZeneca Rare Disease, for its proprietary JUST-AAV ...

AstraZeneca’s rare disease unit Alexion has penned a $825 million deal for adeno-associated virus (AAV) capsids from existing ...

Since its isolation, the biological properties of the adeno-associated virus have been increasingly understood, improving our ability to manipulate and use it as a safe and efficient gene therapy ...

Recently, recombinant adeno-associated virus (rAAV) vectors have been shown to program efficient and stable recombinant gene expression in skeletal muscle and liver in both rodents and primates. 789 ...

The Hopewell site is equipped to support clinical-stage development, process-development, and scaleup for cell and gene therapy programs.

Among them, lentiviruses (LV), adenoviruses (Ad), and adeno-associated viruses (AAV) are the most widely used, accounting for over 80% of approved viral-based gene therapy products. Other viral and ...

Through the program, gene therapies are developed to treat patients with retinitis pigmentosa caused by pathogenic variants ...

Interestingly, our study reveals that SIRT2 is downregulated in JEV infection, SIRT2 genetic deficiency/small molecule inhibition increases viral yield in neuronal cells and mice brains thereby ...