Home > Haematology > ASH 2019 > Late-Breaking Abstracts > BCL11A as a novel target in gene therapy for sickle cell disease

BCL11A as a novel target in gene therapy for sickle cell disease

Presented By
Dr David Williams, Boston Children’s Hospital, USA
ASH 2019
Three adult patients (ages ranging from 21-26 years) with sickle cell disease received an infusion of their own stem cells that were genetically engineered ex vivo to induce them to stop producing harmful sickle haemoglobin and start producing the healthy foetal form of haemoglobin. All 3 patients are doing well and the gene therapy pilot study is extremely promising for future development. Dr David A. Williams (Boston Children’s Hospital, USA) said that the 3 patients, who are now 18, 10, and 9 months post-infusion, are all producing significantly increased amounts of the healthy form of haemoglobin and have so far shown no therapy-related adverse effects beyond those expected with autologous haematopoietic stem cell transplantation [1]. Dr Williams and colleagues hypothesised that knocking down BCL11A with RNA interference would re-induce foetal gamma-globulin expression. They genetically engineered a virus to deliver a gene that...

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