Gene Therapy Articles
Affecting more than 100,000 American children and adults, sickle cell disease is an inherited hemoglobinopathy that results when a single-nucleotide mutation in the ß-globin gene yields an abnormal “sickle” hemoglobin (HbS). Now, these patients may be eligible for a one-time gene therapy that offers the potential of a durable functional cure by eliminating severe VOCs and associated hospitalizations.
Precise, personal treatment is the way of the future. Here's how experts are using genetic sequencing to forge a revolutionary new way of practicing medicine.
Exciting new developments in gene therapy and genome editing show promise for treating a variety of genetic diseases.
New clinical studies show gene therapy may offer a cure for these chronic and expensive diseases in five to 10 years.
The decades-old dream is to correct the factor deficiency through a single infusion of a hemophilia gene therapy that produces steady high protective levels of FVIII or FIX.
Optimism continues to grow about the use of stem cells in regenerative medicine highlighted by hundreds of successful clinical trials.
Potentially curative gene therapy is in the works for hemophilia.
Gene therapy is the one remaining therapeutic option for treating severe combined immunodeficiency by correcting it at its most fundamental genetic level.
