Blood Diseases

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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Mitochondrial DNA sequencing reveals the true origins of cells

April 9, 2019

There are many more cell types than you might think. In your liver, for example, the cells in one section may be different than those in another. But researchers have only a general idea of how stem cells differentiate into broad categories (like “liver”) and divide to make new cells.

Understanding each different cell type – and how it interacts with its neighbors over time – is one of the greatest challenges in biomedical research.

A new study led by HSCI researcher Vijay...

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Cell therapy for all

November 1, 2018

Chad Cowan is on a quest for the universal donor, which would make cell therapy available to populations – not just individuals.

  • Harvard scientist Chad Cowan aims to make off-the-shelf cellular products, and democratize access to new medicines.
  • His lab is working to make cellular therapies that can work for anyone, not just a single patient. 
  • Their ambitious goal is to modify therapeutic cells that can be transplanted into many people, without being rejected.


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One good drug

November 1, 2018

HSCI physician-scientists Daniel Tenen and Li Chai want to turn off a gene to stop cancer without poisoning the patient.

  • The ideal cancer drug would harm cancer cells without hurting normal cells.
  • In their quest to discover one good therapy, HSCI scientists Dan Tenen and Li Chai have zeroed in on SALL4, a gene that is essential for growth in both embryos and cancers.
  • A cellular therapy that deprives cancer cells of SALL4 would stop a third of cancers in their tracks.

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