In contrast to the standard enzyme-replacement therapy, administered from once per 7–14
days to 2–3 times a week in patients with severe hemophilia B, as a result of a single …

Three recent approvals and over 100 ongoing clinical trials make adeno‐associated virus
(AAV)‐based vectors the leading gene delivery vehicles in gene therapy. Pharmaceutical …

Gene therapy has become a realistic option for the treatment of various genetic diseases [1].
It involves the use of a vector (viral/non-viral) to deliver the required transgene for …

Adeno-associated virus (AAV) capsids have shown clinical promise as delivery vectors for
gene therapy. However, the high prevalence of pre-existing immunity against natural …

Photoreceptors, the primary site of phototransduction in the retina, require energy and
metabolites to constantly renew their outer segments. They preferentially consume most …

Impressive achievements in clinical trials to treat hemophilia establish a milestone in the
development of gene therapy. It highlights the significance of AAV-mediated gene delivery to …

Breast carcinoma has one of the highest incidence rates (11.7%), with significant clinical
heterogeneity. Although conventional chemotherapy and surgical resection are the current …

Hemophilic arthropathy (HA) due to repeated bleeding into the joint cavity is a major cause
of morbidity in patients with hemophilia. The molecular mechanisms contributing to this …

Exosome associated Adeno-associated virus (AAV) vectors have emerged as a promising
tool in gene therapy. Recently, we elucidated the role of SUMOylation post-translational …

It is crucial to develop a long-term therapy that targets hemophilia A and B, including
inhibitor-positive patients. We have developed an Adeno-associated virus (AAV) based …