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Columbia scientists use CRISPR gene editing to boost energy production in eye cells

This experimental therapy targets a common metabolic issue in Retinitis Pigmentosa

21-Mar-2024

Key points from article :

Columbia University researchers are exploring how correcting a metabolic problem in light-sensing eye cells could become a universal treatment for Retinitis Pigmentosa (RP).

RP, caused by mutations in over 80 genes, leads to severe vision loss, and current gene therapies only work for a small subset of patients.

Scientists used CRISPR gene editing to target energy production in rod cells (responsible for night vision), hoping to slow the degeneration of both rods and cones (responsible for day vision).

Researchers boosted a process called glycolysis (how cells create energy from glucose) specifically in rod cells, theorizing it would preserve the health and functioning of neighboring cone cells.

This experimental therapy delayed RP progression in mouse models by roughly the human equivalent of 10 years, showing promise for treating multiple forms of RP.

Researchers plan to refine the therapy in larger animal models and further investigate how this metabolic enhancement combats the effects of RP and aging.

The study is published in the journal Cell Reports Medicine.

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Cell Reports Medicine

Open-access journal from Cell Press

Columbia University

Private Ivy League research university in New York City

Topics mentioned on this page:
Vision (health), Gene Therapy
Columbia scientists use CRISPR gene editing to boost energy production in eye cells