Archive for blood disorder
Avoiding a lifetime of injections: Can gene editing cure severe congenital neutropenia?
Fionn Mulrooney, a cheerful 11-month-old, in Plymouth, Massachusetts, has no idea he has a life-threatening genetic disease. Nor does he seem fazed by the daily subcutaneous injections his parents have learned how to give him. And little does he know that cells from his bone marrow are helping scientists develop an innovative gene-editing approach that ... Read More
Arthritis drug reduces rates of acute graft-vs-host disease after bone marrow transplant
The immune-suppressing drug abatacept, currently used for rheumatoid arthritis, could make bone marrow transplant safer, report researchers at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. The phase 2 randomized, multi-center clinical trial, the largest to date, appears in the Journal of Clinical Oncology. Abatacept (brand name ORENCIA) reduced rates of severe, acute graft-versus-host disease ... Read More
Navigating the aplastic anemia journey
Hadas Asgedom is a woman with many titles: daughter, sister of 12, aunt, mother. But, after her own daughter’s journey with aplastic anemia, she may have earned the right to add another — relentless researcher. Hadas and her only child, Asada, live in Tampa, Florida, where Asada had always enjoyed theater rehearsals, soccer, and time ... Read More
A first for CRISPR: Cutting genes in blood stem cells
CRISPR — a gene editing technology that lets researchers make precise mutations, deletions and even replacements in genomic DNA — is all the rage among genomic researchers right now. First discovered as a kind of genomic immune memory in bacteria, labs around the world are trying to leverage the technology for diseases ranging from malaria ... Read More
Supercharged marrow transplant: Zebrafish reveal drugs that aid engraftment
Bone marrow transplantation, a.k.a. stem cell transplantation, can offer a cure for certain cancers, blood disorders, immune deficiencies and even metabolic disorders. But it’s a highly toxic procedure, especially when a closely matched marrow donor can’t be found. Using stem cells from umbilical cord blood banked after childbirth could open up many more matching possibilities, ... Read More
Moving gene therapy into high gear
Gene therapy, still experimental but beginning to enter the clinic, attempts to utilize advanced molecular methods to treat and even reverse genetic diseases. The field started in earnest about 25 years ago and has had many setbacks along the way to its recent earliest successes. International collaboration has been critical. Children’s Hospital Boston is one ... Read More