Publications by authors named "L Krishnamurti"
Sickle cell disease in India: the journey and hope for the future.
Hematology Am Soc Hematol Educ Program
December 2024
Article Synopsis
- India has a high frequency of the sickle hemoglobin (HbS) allele, and though sickle cell disease (SCD) was once considered mild in the country, recent advancements indicate its severity is on par with global standards.
- Clinical manifestations of SCD in India include vaso-occlusive crises and strokes, with affected individuals experiencing complications at a younger age, but improvements in care have led to increased survival into adulthood.
- The Government of India has initiated the National Sickle Cell Anaemia Elimination Mission to enhance screening, treatment, and education efforts, with funding aimed at developing new therapies and technologies to manage SCD.
Disease-modifying therapies are standard of care (SOC) for sickle cell disease (SCD), but hematopoietic cell transplantation (HCT) has curative potential. We compared outcomes prospectively through 2-years after biologic assignment to a Donor or No Donor (SOC) Arm based on the availability of an HLA-matched sibling or unrelated donor (BMTCTN 1503; NCT02766465). A donor search was commenced after eligibility confirmation.
View Article and Find Full Text PDFBIVV003 is a gene-edited autologous cell therapy in clinical development for the potential treatment of sickle cell disease (SCD). Hematopoietic stem cells (HSC) are genetically modified with mRNA encoding zinc finger nucleases (ZFN) that target and disrupt a specific regulatory GATAA motif in the BCL11A erythroid enhancer to reactivate fetal hemoglobin (HbF). We characterized ZFN-edited HSC from healthy donors and donors with SCD.
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