Over the past decade, in vivo gene replacement therapy has significantly advanced,
resulting in market approval of numerous therapeutics predominantly relying on adeno …

The Central Nervous System (CNS) is vulnerable to a range of diseases, including
neurodegenerative and oncological conditions, which present significant treatment …

Gene therapy has evolved into a pivotal approach for treating genetic disorders, extending
beyond traditional methods of directly repairing or replacing defective genes. Recent …

Adeno-associated virus type 2 (AAV2) is a small, non-pathogenic, helper virus-dependent
parvovirus with a single-stranded (ss) DNA genome of approximately 4.7 kb. AAV2 DNA …

Recombinant adeno-associated virus (AAV) vectors have emerged as the preferred platform
for gene therapy of rare human diseases. Despite the clinical promise, host immune …

Objective Progressive retinal atrophy has been described after subretinal gene therapy
utilizing the adeno-associated virus (AAV) vector platform. To elucidate whether this atrophy …

Ocular gene therapy has rapidly advanced from proof-of-concept studies to clinical trials by
exploiting the unique advantages of the eye, including its easy accessibility, relative immune …

With more than 130 clinical trials and 8 approved gene therapy products, adeno-associated
virus (AAV) stands as one of the most popular vehicles to deliver therapeutic DNA in vivo …

Epstein‐Barr virus (EBV) infection is closely associated with the development of various
tumors such as lymphomas and epithelial cancers. EBV has a discrete life cycle with latency …

We sought to engineer mammalian cells to secrete nuclease activity as a step toward
removing the need to purchase commercial nucleases as process additions in …