How Gene Therapy Is Curing Diseases
For patients with genetic disease, recent evolution of and FDA approval for gene therapies are transforming care and turning an ambitious dream into a life-changing reality. But getting affordable treatments to patients safely and efficiently remains a challenge.
Breakthroughs in Gene Therapy
Exciting new developments in gene therapy and genome editing show promise for treating a variety of genetic diseases.
Hemophilia Gene Therapy: Cures May Finally Be at Hand
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.
New Gene Therapy Treatment Offers Possible Cure for SCID-X1
Researchers at St. Jude Children’s Research Hospital have cured infants with X-linked severe combined immunodeficiency (SCID-X1) using gene therapy involving a re-engineered virus.
Gene Therapy for Hemophilia: Edging Closer to Reality
Potentially curative gene therapy is in the works for hemophilia.
Toward the Ultimate Cure: Gene Therapy for Severe Combined Immunodeficiency
Gene therapy is the one remaining therapeutic option for treating severe combined immunodeficiency by correcting it at its most fundamental genetic level.
Using Genomic Data to Personalize Cancer Treatment
Researchers are using genomics research to better understand how genetic variation contributes to human health and disease.
New Gene Therapy Could Be Safe, Effective for Hemophilia B Patients
A new kind of gene therapy tested in animals could be safe and effective for human patients with hemophilia B.
Immune Disorders Genetic Missing Link Found
An international team of researchers has identified a gene that may be a missing link between overactive and underactive immune activity, and that may also play a key role in autoimmune diseases.
Breaking the Cancer Code
Scientific breakthroughs using gene therapy are allowing physicians to take a “precision medicine” approach to treating cancer — with promising results.