GEN

Delta Force: Scientists Promote Novel Globin Genetic Therapy for Sickle Cell Disease

Mandy Boontanrart, PhD, a postdoc at ETH Zurich in Switzerland, has a particularly personal motivation for her research on a novel genetic therapy for sickle cell disease (SCD). She is a carrier of a ...
Business Wire

Graphite Bio Presents Preclinical Gene Replacement Data for GPH102 for Beta-thalassemia at the ASGCT 25th Annual Meeting

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage, next-generation gene editing company harnessing the power of high-efficiency precision gene repair to ...

One-time gene therapy could end lifelong transfusions for rare blood disease

Thanks to in-utero blood transfusion technology, what was once a fatal diagnosis in the womb can now result in live births. However, this medical advancement created a new challenge: a growing ...
The American Journal of Managed Care

Preclinical Study of Gene Therapy Vector for SCD, Beta-Thalassemia Shows Promise

An engineered lentivirus was created to avoid some of the drawbacks associated with current gene therapy vectors for sickle cell disease (SCD) and beta-thalassemia. Researchers reported Friday that a ...
GEN

Base Editing Beats Other Genome Editing Strategies for Treating Sickle Cell Disease

Base editing—the genome editing technique that alters base pairs using a nuclease-deficient Cas9 fused to a deaminase—was found to restart fetal hemoglobin expression in sickle cell disease (SCD) ...
Medscape

Gene Therapy Frees β-Thalassemia Patient From Transfusions for More Than 2 Years

September 16, 2010 — Treating β-thalassemia with gene therapy has enabled a young adult patient who received his first transfusion at age 3 years to live without transfusions for more than 2 years.
The New Yorker

A Stunning Gene-Therapy Breakthrough in the Fight Against Beta Thalassemia, a Devastating Blood Disease

In 1976, my final year of medical school, I travelled abroad and spent several months working in the hematology clinic at Hadassah Hospital, in Jerusalem. Every day, I attended to children and ...
JSTOR Daily

Correction of a Splice-Site Mutation in the Beta-Globin Gene Stimulated by Triplex-Forming Peptide Nucleic Acids

Splice-site mutations in the beta-globin gene can lead to aberrant transcripts and decreased functional beta-globin, causing beta-thalassemia. Triplex-forming DNA oligonucleotides (TFOs) and peptide ...
Healio

Gene therapy potentially curative for patients with transfusion-dependent beta-thalassemia

Please provide your email address to receive an email when new articles are posted on . Thompson described beta-thalassemia as a severe chronic inherited blood disorder due to one of many mutations in ...
JSTOR Daily

Introduction and Expression of a Rabbit $\beta $-globin Gene in Mouse Fibroblasts

The cloned chromosomal rabbit $\beta $-globin gene has been introduced into mouse fibroblasts by DNA-mediated gene transfer (transformation). In this report, we examine the expression of the rabbit ...
Science Daily

Researchers create new viral vector for improved gene therapy in sickle cell disease

Researchers have developed a new and improved viral vector -- a virus-based vehicle that delivers therapeutic genes -- for use in gene therapy for sickle cell disease. In advanced lab tests using ...