We present an overview of clinical trials involving gene editing using clustered interspaced
short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription …

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 …

Hemophilia B (HB) is a life-threatening inherited disease caused by mutations in the FIX
gene, leading to reduced protein function and abnormal blood clotting. Due to its monogenic …

Gene therapy represents an attractive alternative to treat hemophilia B. Here we established
three hepatocyte-derived cell lines based on Huh7, PLC/PRF/5, and Hep3B cells stably …

Genome editing techniques are considered to be one of the most challenging yet efficient
tools for assisting therapeutic approaches. Several studies have focused on the …

Congenital coagulopathies have, throughout the history of medicine, been a focus of
scientific study and of great interest as they constitute an alteration of one of the most …

Introduction Currently, a few in vivo gene replacement therapies are commercially available,
with many in clinical development for the treatment of some inherited monogenic diseases …

In this issue of Blood, Wang et al report correction of the bleeding phenotype in newborn
and adult factor IX (FIX) knockout mice through in vivo gene editing mediated by clustered …

Since observations that CRISPR nucleases function in mammalian cells, many strategies
have been devised to adapt them for genetic engineering. Here, we investigated self-cutting …

Since the middle of the last century, there have been amazing therapeutic advances for
hemophilia such as the development of plasma-derived products and bioengineered …