The Rutgers Cancer Institute recently became a designated gene therapy treatment center for sickle cell anemia and beta-thalassemia, joining a small group of medical centers that currently provide ...

The parents of a seven-year-old boy who died in a clinical trial have spoken of their "indescribable" pain. Ender Nihat, from Essex, suffered organ failure after receiving chemotherapy during ...

In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...

This writer digs into the CRISPR gene-editing space, asking whether one particular penny stock is worth taking a punt on in ...

India is known as the “Thalassemia capital of the world.” It has the highest number of patients with thalassemia major in the world. India has around 50 million (5 crore) carriers of the ß ...

My brothers and I compare our genetic analyses and what diseases we are genetically at risk for and what conditions we ...

Like other developers of recently approved gene therapies ... received agency authorization to treat transfusion-dependent beta thalassemia in patients 12 years and older. As of mid-October ...

Doctors harvest stem cells from the patient’s bone marrow and then send them off to a lab, where CRISPR is used to turn up the production of another, functional version of haemoglobin. This version is ...

Bluebird also faced pushback from European payers after pricing its gene therapy for blood disorder beta thalassemia, called Zynteglo, at $1.8 million per patient. The company withdrew the ...

Building a gene editor took months. With CRISPR even high-schoolers can get hold of editing systems in the time it takes to order RNA sequences online and have them shipped by FedEx. In a short ...

This medicine uses the gene-editing tool, namely, clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) (CRISPR-Cas9), to treat β-thalassemia and ...