In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic
disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served …

Adeno-associated viruses (AAV) have emerged as the lead vector in clinical trials and form
the basis for several approved gene therapies for human diseases, mainly owing to their …

Adeno-associated viral (AAV) vector gene therapy has shown promise as a possible cure for
hemophilia. However, immune responses directed against AAV vectors remain a hurdle to …

Introduction Hemophilia comprises a group of X-linked hemorrhagic disorders that result
from a deficiency of coagulation factors. The disorder affects mainly males and leads to …

Gene therapy directly targets mutations causing disease, allowing for a specific treatment at
a molecular level. Adeno-associated virus (AAV) has been of increasing interest as a gene …

Given the recent success of gene therapy modalities and the growing number of cell and
gene-based therapies in clinical development across many different therapeutic areas, it is …

The therapeutic landscape for people living with hemophilia A (PwHA) has changed
dramatically in recent years, but many clinical challenges remain, including the development …

Hemophilia A (HA) is a rare bleeding disorder caused by deficiency/dysfunction of the FVIII
protein. As current therapies based on frequent FVIII infusions are not a definitive cure, long …

Introduction: Rare genetic diseases affect millions of people worldwide. Most of them are
caused by defective genes that impair quality of life and can lead to premature death. As …

Hypertension is a major contributor to the global burden of disease. Unfortunately,
hypertension is controlled in less than one-fifth of patients worldwide due to either failure to …