Harvard Stem Cell Institute
Progress Report: March 2018 
Modeling autism with patient stem cells
Purkinje neuron
Autism spectrum disorder (ASD) is a complex disorder that, until now, could not be readily studied in the lab. A recent study led by Mustafa Sahin, HSCI Affiliate Faculty member, describes a new way to model and investigate ASD.

What they did : Using stem cells from patients with tuberous sclerosis complex, a disease associated with ASD, the researchers developed a method to make the specific type of neuron thought to be affected in ASD.

What they found : HSCI researchers found that these patient-derived Purkinje neurons had structural defects, and one overactive biological pathway. When they treated the cells with rapamycin -- an approved drug that inhibits the pathway -- the researchers observed that cell function improved.

Why it matters : Looking ahead, these Purkinje neurons will be a good model for studying ASD further and testing potential drugs.
Muscle regeneration research collaboration with Frequency Therapeutics
muscle regeneration
The biotechnology company Frequency Therapeutics has announced a research collaboration with the U.S. Army and two HSCI Principal Faculty members: Lee Rubin and Amy Wagers.
 
The goal : The collaboration aims to develop drugs that activate muscle satellite cells, which then differentiate into muscle cells.

HSCI's contribution : The research leverages Rubin's expertise in drug discovery and neuromuscular disease, and Wagers' expertise in skeletal muscle biology and the isolation of muscle satellite cells.

Why it matters : This work has the potential to improve muscle repair following injury or disease.
Vijay Sankaran receives Drukier Prize in Children's Health Research
Sankaran award
Weill Cornell Medicine recently announced that Vijay Sankaran, HSCI Principal Faculty member, has been awarded the Gale and Ira Drukier Prize in Children's Health Research. The prize honors an early-career pediatrician whose research has made important contributions to improving children's health. 

Sankaran was recognized for his research on hereditary red blood cell disorders, including sickle cell disease and thalassemia. He identified a gene that regulates levels of fetal hemoglobin, a molecule that transports oxygen around the body. 

Sankaran is currently developing gene therapy approaches to improve fetal hemoglobin levels in patients.

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