Blood Diseases Program

HSCI scientist awarded grant to develop gene therapy for HIV

September 7, 2020

HSCI co-director David Scadden is part of a multi-institutional, cross-disciplinary team that has been awarded a $14.6 million grant from the National Institutes of Health. The team is using gene and cell therapy approaches to develop a potential cure for HIV.

The researchers will combine gene editing against HIV with technologies for safer and more effective blood stem cell transplants. Such transplants, also known as bone marrow transplants, are currently used for severe blood cancers. They renew a patient’s immune system, which can be damaged by cancer therapies, by...

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Tracking an organism’s development, cell by cell

May 29, 2020

HSCI scientists develop CRISPR-based tool for lineage tracing

A new mouse model allows scientists to track every cell in the body, from the embryo stage until adulthood. The system, published in the journal Cell, could yield a greater understanding of development, aging, and disease.

“The dream of many developmental biologists for decades is a way to reconstruct every single...

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HSCI scientist receives Harrington Prize for sickle cell research

May 18, 2020

HSCI principal faculty member Stuart Orkin, M.D. is the recipient of the 2020 Harrington Prize for Innovation in Medicine. The award, established in 2014 by the Harrington Discovery Institute and the American Society for Clinical Investigation (ASCI), honors physician-scientists who have moved science forward with achievements notable for innovation, creativity and potential for clinical application.

Orkin is the David G. Nathan Distinguished Professor of Pediatrics at Harvard Medical School. He is being recognized for breakthrough discoveries about red blood cells that offer...

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Potential drug treatments for telomere diseases

April 24, 2020

HSCI researchers have identified potential drugs to treat telomere diseases such as dyskeratosis congenita (DC), in which cells age prematurely. The researchers tested over 100,000 compounds on patient stem cells, and identified several drugs that restored  the cells’ telomeres — the protective caps on the ends of chromosomes that regulate how cells age.

“We envision these to be a new class of oral medicines that target stem cells throughout the body,” said Suneet Agarwal, an HSCI principal faculty member at Boston Children’s Hospital. “We expect restoring telomeres in...

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