A major challenge in human genetics is understanding which parts of the genome drive specific traits or contribute to disease risk. This challenge is even greater for genetic variants found in the 98% of the genome that does ...
May 4, 2023
0
33
Researchers at Children's Hospital of Philadelphia (CHOP) have discovered a new part of the regulatory cascade responsible for silencing fetal hemoglobin in adult cells. The findings, published today in Nature Genetics, refine ...
Aug 8, 2022
0
53
In a late-breaking abstract presented at the European Hematology Association (EHA) Congress, a group of researchers that includes Children's Hospital of Philadelphia (CHOP) presented new data on an investigational therapy ...
Jun 14, 2022
0
8
The genetic blood disorders sickle cell disease and beta-thalassemia are caused by errors in the genes for hemoglobin, a protein in red blood cells that carries oxygen from the lungs to tissues throughout the body. In utero, ...
May 26, 2022
0
42
A series of laboratory studies is underway in the United States to improve gene therapy worldwide for sickle cell disease, a complex and sometimes deadly heritable blood disorder that dramatically affects the structure and ...
For the millions of people worldwide who have sickle cell disease, there are only a few treatment options, which include risky bone marrow transplants, gene therapy or other treatments that address a subset of symptoms. Today, ...
Apr 9, 2021
0
51
Scientists are seeing promising early results from the first studies testing gene editing for painful, inherited blood disorders that plague millions worldwide, especially Black people.
Dec 5, 2020
0
263
"Mothering" is synonymous with "nurturing," probably because moms start providing for their kids even before they're born.
May 5, 2020
0
2
A pilot gene therapy treatment for sickle cell disease, restoring patients' ability to make fetal hemoglobin, has produced good results in the first three patients to receive it. Investigators at Boston Children's Hospital ...
Jan 10, 2020
0
2
In a paper published in the July 31 issue of Science Translational Medicine, researchers at Fred Hutchinson Cancer Research Center used CRISPR-Cas9 to edit long-lived blood stem cells to reverse the clinical symptoms observed ...
Jul 31, 2019
0
87
