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What's new in December 2020

Reversing aging in the eye

Axons of regenerating neurons.
HSCI researchers led by Zhigang He and Bruce Ksander are part of a team that has developed a gene therapy to reverse age-related vision loss.
  • What they did: The researchers developed a therapy using a combination of three genes, which erase the chemical patterns on DNA that accumulate as cells age.
  • What they found: In mouse models, the treatment successfully regenerated injured optic nerves and reversed the age-related vision loss that occurs with glaucoma.
  • Why it matters: If replicated through further studies, the approach could pave the way for therapies to promote tissue repair across various organs and reverse age-related diseases in patients.

A tool to investigate the function of autism spectrum disorder genes

Gene editing in the brain.
HSCI researchers led by Paola Arlotta are part of a team that has developed a technology to investigate the function of many different genes in many different cell types at once, in a living organism. They applied the method to study dozens of genes that are associated with autism spectrum disorder.
  • What they did: The researchers combined two powerful genomic technologies:  CRISPR gene editing to make changes in 35 different genes linked to autism spectrum disorder risk, and single-cell RNA sequencing to analyze responses in the developing mouse brain.
  • What they found: By looking at data from over 40,000 individual cells, the researchers found that neurons and glia — the non-neuronal cells in the brain — are affected by different sets of the risk genes.
  • Why it matters: This large-scale tool will help researchers to pinpoint the mechanisms of how autism spectrum disorder risk genes can lead to disease.

Missed signals: A new source of biological variation

Red blood cells.
HSCI’s Leonard Zon has identified a new source of biological variation: a gene’s ability to react to chemical signals from the outside the cell.
  • What they did: The researchers studied genetic variants that are associated with specific red blood cell traits, such as size and hemoglobin concentration.
  • What they found: Many of the variants were located in DNA sequences that changed how the genes could respond to signals from outside the cell.
  • Why it matters: The findings could apply to other tissues or organs in the body and may help explain why some people are more or less susceptible to disease, why organs function differently, and why some people respond more to drugs than others.
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