One of the well-known X-linked genetic disorders is hemophilia, which could be hemophilia
A as a result of a mutation in the F8 (factor VIII) gene or hemophilia B as a result of a …

Most rare diseases (RDs) encompass a diverse group of inherited disorders that affect
millions of people worldwide. A significant proportion of these diseases are driven by …

Adeno-associated viruses (AAVs) have emerged as a prominent family of vectors for gene
delivery, providing therapeutic options to diseases once deemed incurable. At the same …

Genome or prime editing has become a promising tool for the treatment of hereditary
disorders affecting the inner retina, such as dominant optic neuropathies. In vivo delivery of …

Adeno-associated virus (AAV)-based gene therapy has become one of the key directions of
modern translational medicine geared towards treatment of hereditary disorders by means …

Gene therapy and its role in the medical field have evolved drastically in recent decades.
Studies aim to define DNA-based medicine as well as encourage innovation and the further …

Recombinant adeno‐associated virus (rAAV) has emerged as an attractive gene delivery
vector platform to treat both rare and pervasive diseases. With more and more rAAV‐based …

Huntington's Disease (HD) is a debilitating neurodegenerative disorder characterized by
progressive motor dysfunction, cognitive decline, and psychiatric symptoms, resulting from …

Hemophilia is the most frequent severe genetic haemorrhagic condition. Hemophilia A and
B are caused by a lack or dysfunction of the factor VIII and factor IX proteins, respectively …

Adeno-associated viruses (AAVs) have emerged as a foundational vector for gene therapy
delivery. Recognized for their remarkable ability to deliver a transgenic payload into both …