Todd-Haenfler

CRISPR Reactivation of the Fragile X Gene

Using CRISPR to reactivate FMRP, “We are trying to target the first event that goes wrong in Fragile X syndrome”, says FRAXA Investigator, Dr. Peter Todd.

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Dr. Peter Todd

Targeted Transcriptional Reactivation of FMR1 in Fragile X Syndrome Stem Cells

FRAXA funded Dr. Peter Todd to use CRISPR to reactivate FMR1. Published results confirmed restored gene expression, a big step toward disease-modifying therapy.

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Dr. Haenfler and Dr. Todd

University of Michigan researcher Peter Todd, MD, PhD, Aims to Selectively Turn the Fragile X Gene Back on in Human Cells

University of Michigan scientists Peter Todd, MD, PhD, and postdoctoral fellow Jill Haenfler, Ph.D., are adapting CRISPR to reactivate the FMR1 gene to reverse Fragile X syndrome.

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FRAXA Funded Research

Current Research Grants (38)