University of Connecticut
March 23, 2021
UConn professor of physiology and neurobiology Daniel Mulkey has received a $2.1 million grant from the NIH to investigate the cellular and molecular basis for disordered breathing in Rett syndrome. This project is part of a collaboration with Michelle Olsen, associate professor of neurobiology ...
March 15, 2021
UVA’s Sanchita Bhatnagar, PhD, discovered that tiny bits of RNA, called microRNAs, play an important role in Rett, a rare genetic disorder that can impair children’s ability to speak, move and even breathe.
Texas Children's Hospital
ASO as a feasible therapy to treat MECP2 duplication disorder
March 4, 2021
A study by Dr. Zoghbi provides experimental evidence that supports the use of antisense oligonucleotides as a feasible strategy to treat MECP2 Duplication Syndrome. Funding for this work came from the MECP2 Duplication Syndrome Fund at RSRT.
Rett Syndrome Awareness on Anderson Coooper's Full Circle
March 2, 2021
Four girls with Rett Syndrome and their moms discuss what they are grateful for in the "For Goods" section of "Full Circle"
March 1, 2021
Altering a protein linked to Rett syndrome so that it cannot bind to a particular type of DNA tag may explain the syndrome’s delayed onset in people.
The Washington Post
They thought they’d be near the front of the line for the vaccine. Now, they don’t know where they stand.
February 13, 2021
The long wait for vaccination has left many people across the country feeling anxious, desperate even. But for the young and disabled, and for the people who care about them, the realization of where they fall on priority lists has also left them feeling disregarded and discarded.
February 1, 2021
Taysha Gene Therapies, Inc. has highlighted its strategic priorities and provided a business outlook for 2021.
Neurogene and University of Edinburgh Announce Research Collaboration to Advance Next Generation Gene Therapies
January 19, 2021
Neurogene Inc. has announced a collaboration to advance development of multiple platform approaches designed to enable next generation gene therapies.
January 14, 2021
Two unpublished studies detail improved methods for delivering gene therapies to the brain: One involves a type of stem cell that can produce gene-altering proteins on-site; the other taps an engineered virus to target neurons efficiently and noninvasively.
The New York Times
December 19, 2020
Clusters of living brain cells are teaching scientists about diseases like autism. With a new finding, some experts wonder if these organoids may become too much like the real thing.
October 14, 2020
A gene therapy for Angelman syndrome stands at the forefront of efforts to treat autism-linked conditions that stem from single genes.
October 3, 2020
October is Rett Syndrome Month. It's a rare and very challenging disease that affects just one in 10,000 girls. It's a tough go, but there is hope. Tim Freeman from the Rett Syndrome Research Trust tells us more.
August 27, 2020
Problems with the formation of DNA-filled droplets that control gene expression may contribute to Rett syndrome, a new study suggests.
Taysha Gene Therapies
Taysha Gene Therapies Announces Oversubscribed $95 Million Series B Financing to Bolster Initial Clinical Studies in GM2 Gangliosidosis and Advance Pipeline of Gene Therapies for Monogenic CNS Disease in Both Rare and Large Patient Populations
August 5, 2020
Taysha Gene Therapies, a patient-centric gene therapy company with a mission to eradicate monogenic CNS disease, today announced that it has closed an oversubscribed $95 million Series B financing with a premier syndicate of life science investors, led by Fidelity Management & Research ...