Many currently incurable forms of blindness affecting the retina have a genetic etiology and
several others, such as those resulting from retinal vascular disturbances, respond to …

Ocular gene therapy has been extensively explored in recent years as a therapeutic avenue
to target diseases of the cornea, retina and retinal pigment epithelium (RPE). Adeno …

Adeno-associated virus (AAV) vectors are attractive for gene delivery-based therapeutics,
but data from recent clinical trials have indicated that AAV capsids induce a cytotoxic T …

We investigated the transduction of HEK293T cells permissive to adeno-associated virus
serotype 8 (AAV8) to understand the mechanisms underlying its endocytic processing …

A drawback of gene therapy using adeno-associated virus (AAV) is the DNA packaging
restriction of the viral capsid (< 4.7 kb). Recent observations demonstrate oversized AAV …

As a consequence of their well‐defined nanostructure and intrinsic bioactive functionality,
virus‐based nanoparticles have shown promise for mediating gene delivery. Adeno …

The unfolded protein response (UPR) is a stress-induced cyto-protective mechanism elicited
towards an influx of large amount of proteins in the endoplasmic reticulum (ER). In the …

US8501167B2 - Compositions and methods for targeted ablation of mutational escape of
targeted therapies for cancer - Google Patents US8501167B2 - Compositions and methods …

Heart transplant gene therapy requires vectors with long‐lasting gene expression, high
cardiotropism, and minimal pathological effects. Here, we examined transduction properties …

ABSTRACT The Adeno-Associated Virus has moved to the forefront as a vector for human
gene therapy. Vectors have been constructed with AAV to repair many genetic deficiencies …