Rocket's first two clinical programmes using LVV-based gene therapy are for the treatment of Fanconi Anemia
, a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, and Leukocyte Adhesion Deficiency-I (LAD-I), a serve pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal.
Focusing on this growth pathway may finally open the door to better therapies for children with Fanconi anemia
signaling network regulates the spindle assembly checkpoint.
The immunocompromised child suffering from Fanconi anemia
developed a jejunal fistula after removal of the feeding tube (Table 1).
The report provides comprehensive information on the therapeutics under development for Fanconi Anemia
, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.
Auerbach, "Fanconi anemia
and its diagnosis," Mutation Research, vol.
Ocular and orbital manifestations of the inherited bone marrow failure syndromes: Fanconi anemia
and dyskeratosis congenita.
Better posttransplant outcome with fludarabine based conditioning in multitransfused fanconi anemia
patients who underwent peripheral blood stem cell transplantation.
(FA) is a rare chromosome instability syndrome that predisposes to bone marrow failure, developmental abnormalities, and a high risk for the development of cancer, such as hematological malignancies, solid tumors of the head and neck region, and gynecological tumors (2-5).
Comparative study of Fanconi anemia
in children of different ethnic origin in South Africa.
He also suffers from Fanconi anemia
, a rare disease that often leads to cancer.
(3.) Green AM, Kupfer GM: Fanconi anemia
. Hematol Oncol Clin North Am 23(2):193, 2009