UCSF Benioff Children's Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...
Current gene therapies to treat sickle cell disease are complex, time-consuming, and are sometimes linked to serious side effects like infertility or blood cancer. To address these challenges, Johns ...
New results from a clinical trial show promising outcomes for a gene-edited treatment for severe sickle cell disease, a ...
UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...
Sickle cell thalassemia occurs when you inherit both a sickle cell gene mutation and a thalassemia gene mutation. Symptoms are similar to other types of sickle cell disease but can vary depending on ...
A publicly funded consortium of University of California (UC) researchers and clinicians is one big step closer to realizing a decade-long plan to launch a CRISPR-based gene-editing trial for sickle ...
The National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending a CRISPR gene editing-based treatment for severe sickle cell disease (SCD). The regulator said ...
Sickle cell disease is a hereditary condition, which means a person inherits it from their biological parents. The condition occurs when a person inherits two copies of the hemoglobin beta (HBB) gene ...
If you or someone you know has sickle cell disease (SCD), you may have heard of Lyfgenia, one of two gene therapies approved for this condition. Gene therapies are the only treatments for SCD that can ...
Dr Annarita Miccio told the ELRIG meeting in March 2026 that gene therapy for sickle cell disease and beta-thalassaemia has ...
This article is part of “Innovations In: Sickle Cell Disease,” an editorially independent special report that was produced with financial support from Vertex Pharmaceuticals. Sickle cell disease was ...
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