SCD is an inherited blood disease that causes abnormal hemoglobin function due to a mutation in the gene that encodes the hemoglobin β s peptide chain.
affects the lives of millions of patients around the world, with 300,000 newborns suffering from the disease each year.
the genetic pathogenesies of SCD have been clarified, it was only in recent years that the biomedical community gained access to tools to treat the disease by correcting genes.
gene therapy or gene-editing therapy, which is currently in the clinical stage, requires the removal of cells from the patient's body, the genetic engineering of these cells in vitro, and then the transmission of the modified cells back.
Although the therapy has shown remarkable efficacy recently, the equipment used to make this gene therapy usually does not exist in the most widespread areas of SCD, so the vast majority of patients do not have access to the gene therapy that could change their lives.
plans to develop an in vivo gene therapy for SCD.
It may only require a one-time direct injection into the patient and does not require genetically engineered modification of cells in vitro, thus avoiding many in vitro gene therapy-related hospitalization processes and specialized laboratory equipment.
to promote research, the Gates Foundation will fund the Novarma Institute for Biomedical Research (NIBR) to support a dedicated research team in this direction.
"the existing gene therapy for SCD is difficult to spread on a large scale, and most patients face challenges in the accessability of treatment.
, NIBR President, said: "This is a challenge that needs to be addressed by solidarity.
we are pleased to have the support of the Bill and Melinda Gates Foundation to address this global unmet medical need.
: This article is intended to introduce medical and health research, not treatment options recommended.
if you need guidance on treatment options, visit a regular hospital.
resources: novartis and the Bill and Melinda Gates Foundation to collaborate to discover and develop an accessible in vivo the generapy for sickle cell disease. Retrieved February 17, 2021, from