Blood Diseases

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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