Recombinant adeno-associated viruses (AAVs) have emerged as promising gene delivery
vehicles resulting in three US Food and Drug Administration (FDA) and one European …

Regulatory T cells are critical for maintaining immune tolerance. Recent studies have
confirmed their therapeutic suppressive potential to modulate immune responses in organ …

Recombinant adeno-associated virus (AAV) is a promising delivery vehicle for in vivo gene
therapy and has been widely used in> 200 clinical trials globally. There are already several …

Heterotopic ossification is the most disabling feature of fibrodysplasia ossificans
progressiva, an ultra-rare genetic disorder for which there is currently no prevention or …

Chimeric antigen receptor (CAR) T cell therapy, in which a patient's own T lymphocytes are
engineered to recognize and kill cancer cells, has achieved striking success in some …

Adeno-associated viruses (AAVs) are being developed as gene therapy vectors due to their
low pathogenicity and tissue tropism properties. However, the efficacy of these vectors is …

The number of approved or investigational late phase viral vector gene therapies (GTx) has
been rapidly growing. The adeno-associated virus vector (AAV) technology continues to be …

Introduction Altering the human genetic code has been explored since the early 1990s as a
definitive answer for the treatment of monogenic and acquired diseases which do not …

As the clinical experience in adeno-associated viral (AAV) vector-based gene therapies is
expanding, the necessity to better understand and control the host immune responses is …

Ophthalmic autoimmune and autoinflammatory conditions cause significant visual morbidity
and require complex medical treatment complicated by significant side effects and lack of …