Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure and ...

Jasper Therapeutics, Inc. JSPR dosed first patient in the Phase 1/2 clinical study of its drug candidate JPS191 as a conditioning agent in the treatment of Fanconi Anemia. The company has sponsored ...

A Stanford-led team has replaced toxic pre-transplant chemotherapy with a targeted antibody, allowing children with Fanconi ...

Results from a French study of a cohort of individuals with the inherited rare disease Fanconi anemia shed light on how some people with this condition go on to develop secondary leukemia. Writing in ...

Fanconi anaemia (FA) is a rare inherited disorder characterised by genomic instability, progressive bone marrow failure, and a predisposition to malignancies. Haematopoietic stem cell transplantation ...

Gene therapies could have a major impact, although the cost and complexities could inhibit their wide rollout, study authors said. New advancements in gene therapy and hematopoietic stem cell ...

Fanconi anemia is a genetically and phenotypically heterogeneous autosomal recessive disorder characterized by congenital malformations, progressive marrow failure and predisposition to malignancy.1 ...

Fanconi anemia is an aggressive, life-threatening disorder. Most individuals living with this rare genetic condition, characterized by bone marrow failure and cancer predisposition, survive into ...

The rare disease Fanconi anemia is an inherited bone marrow disorder linked to birth defects that leads to failure of bone marrow, the spongy material inside the bones where stem cells develop.

MINNEAPOLIS/ST. PAUL (2/26/2024) — Published in the American Journal of Medical Genetics, a University of Minnesota Medical School study found that it is normal for children with Fanconi anemia to be ...