For people living with rare genetic diseases – many of which can be debilitating or life-threatening – the need for innovative treatments is urgent. Only 5% of the 7,000 known rare diseases ...

A key bottleneck in the field has been the limited availability of safe, selective and effective methods ... gene therapy products use an adeno-associated virus (AAV) as their gene delivery ...

Researchers are enhancing the best features of AAV capsids and overcoming their limitations to accelerate gene-based therapies.

International collaborations, refined gene-editing protocols, and targeted immunologic maneuvers keep the cancer gene therapy ...

In addition, delivery via the vascular route could be exploited to access primary or metastatic lung tumors. These practical considerations favor the development of gene therapy strategies for ...

Twenty years later, she innovates IPEX treatments using gene therapy strategies that edit the mutated FOXP3 in patients’ own immune and stem cells. Medicine and research were always linked for me; I ...

The study did not draw conclusions about past, ongoing or future gene therapy research that relied upon these methods for measuring AAV vectors. It also did not make any policy or regulatory ...

Gene therapy could potentially cure ... cells safely and effectively. Existing methods of engineering one of the most commonly used gene-delivery vehicles, adeno-associated viruses (AAV), are ...

NIS gene delivery into medullary and follicular ... which allows the clinician to pursue therapeutic strategies, including gene therapy, that might ablate normal as well as malignant thyroid ...

BENGALURU: Bengaluru-based Narayana Nethralaya is developing gene therapy solutions that could drastically reduce the cost of ...