Todd-Haenfler

CRISPR Reactivation of the Fragile X Gene

December 6, 2017

“We are trying to target the first event that goes wrong in Fragile X syndrome”, says Todd, “One reason our previous attempts to develop treatments for Fragile X syndrome have failed is that they’ve tried to target the downstream effects of losing the Fragile X protein. The protein does many things… bypassing all the functions that it normally takes care of has proven difficult from a pharmacologic perspective.”

Read More »
Dr. Peter Todd

Targeted Transcriptional Reactivation of FMR1 in Fragile X Syndrome Stem Cells

September 23, 2017

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

Read More »
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

June 13, 2016

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.

Read More »

Categories

FRAXA Funded Research

Current Research Grants (37)