The first CRISPR treatment for blood diseases is showing benefits
The duo of CRISPR Therapeutics and Vertex Pharmaceuticals said this morning that their genome-editing treatment for a pair of blood disorders is showing promise in an early-stage trial.
As STAT’s Sharon Begley and Adam Feuerstein report, two people have been treated with the therapy, and each has been free from blood transfusions and disease symptoms nine months after dosing. One has sickle cell disease and the other has beta thalassemia.
The CRISPR-based therapy, still in the early days of development, is part of a promising wave of potential treatments for inherited blood disorders.
“For decades, we knew about the sickle cell disease mutation but we didn’t know about other genes [involved in the disease] and we didn’t have the necessary tools for genetic correction” of blood-making stem cells, said Dr. Mitchell Weiss of St. Jude Children’s Research Hospital, a leader in the genetics of sickle cell who is not involved in the clinical trial. “Now we have a confluence of scientific understanding and technology that can come together to make things happen.”
Read more.
As STAT’s Sharon Begley and Adam Feuerstein report, two people have been treated with the therapy, and each has been free from blood transfusions and disease symptoms nine months after dosing. One has sickle cell disease and the other has beta thalassemia.
The CRISPR-based therapy, still in the early days of development, is part of a promising wave of potential treatments for inherited blood disorders.
“For decades, we knew about the sickle cell disease mutation but we didn’t know about other genes [involved in the disease] and we didn’t have the necessary tools for genetic correction” of blood-making stem cells, said Dr. Mitchell Weiss of St. Jude Children’s Research Hospital, a leader in the genetics of sickle cell who is not involved in the clinical trial. “Now we have a confluence of scientific understanding and technology that can come together to make things happen.”
Read more.
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