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CRISPR Gene Editing Delivers Promise for Sickle-Cell Disease, Beta Thalassemia

CRISPR Gene Editing Delivers Promise for Sickle-Cell Disease, Beta Thalassemia
CRISPR Therapeutics CEO Samarth Kulkarni, PhD
CRISPR Therapeutics and Vertex Pharmaceuticals have reported a consistent and sustained positive response in 10 patients treated for a pair of blood disorders sickle-cell disease (SCD) and beta thalassemia with their CRISPR-Cas9 gene-edited therapy CTX001 in a pair of Phase I/II trials. These are the first clinical studies of a CRISPR gene-editing candidate sponsored by U.S. companies.
According to data published last Saturday in the
New England Journal of Medicine (NEJM) and presented Sunday at the annual American Society of Hematology (ASH) Meeting and Exposition, all seven patients with transfusion-dependent beta thalassemia (TDT) including three who have either a severe or b0/b0 genotype were transfusion independent at the last follow-up in the TDT trial, which is known as CLIMB-111 (NCT03655678). ....

United States , Davida Williams , David Altshuler , Samarth Kulkarni , Eric Topol , Michaelj Yee , Haydar Frangoul , Fyodor Urnov , Selim Corbacioglu , Leland Fikes , University Of California , Us Centers For Disease , Innovative Genomics Institute At University Of California , American Society Of Hematology , Healthcare Tristar Centennial Medical Center , Innovative Genomics Institute , Sarah Cannon Research Institute , Vertex Pharmaceuticals , National Public Radio , Scripps Research Translational Institute , World Health Organization , European Commission , Harvard Medical School , University Children Hospital Regensburg , European Medicines Agency , New England Journal ,

Two trials show promising results with gene therapies that target sickle-cell anemia

Two trials show promising results with gene therapies that target sickle-cell anemia
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Haydar Frangoul , Ericab Esrick , Harvard Medical School , Farber Cancer Institute , Sciencex Network , New England Journal , Farber Cancer , Sickle Cell Disease , Transcriptional Genetic Silencing , Treat Sickle Cell Disease , ஹார்வர்ட் மருத்துவ பள்ளி , ஃபார்‌பர் புற்றுநோய் நிறுவனம் , புதியது இங்கிலாந்து இதழ் , ஃபார்‌பர் புற்றுநோய் , அரிவாள் செல் நோய் , உபசரிப்பு அரிவாள் செல் நோய் ,