Rett Syndrome Awareness on Anderson Coooper's Full Circle
Four girls with Rett Syndrome and their moms discuss what they are grateful for in the "For Goods" section of "Full Circle"
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.
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.
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
Neurogene Inc. has announced a collaboration to advance development of multiple platform approaches designed to enable next generation gene therapies.
Two unpublished studies detail improved methods for delivering gene therapies to the brain: One involves a type of stem cell that can produce gene-altering proteins on-site;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
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.
A gene therapy for Angelman syndrome stands at the forefront of efforts to treat autism-linked conditions that stem from single genes.
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.
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
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 ...
Biotech Vico Therapeutics raises $31 million (€27 million) in Series A financing round to advance therapies for rare central nervous system diseases
Vico Therapeutics, a Leiden, the Netherlands, based biotech company focusing on the development of RNA modulating therapies for rare neurological disorders, today announced that it has raised $31 million (€27 million) in a Series A financing round.
Scientific first: RNA repair shows promise in reversing mutations underlying a neurological disorder
OHSU study first to use programmable RNA editing to repair gene that contributes to Rett syndrome.
The coronavirus pandemic has stretched hospital resources beyond their capacity, disrupted the delivery of care, and drained providers of billions of dollars of revenue for canceled elective procedures, office visits, and tests.
The world's largest brain research prize is Danish and is awarded by the Lundbeck Foundation. Each year, we award 10 million DKK (approx. 1,3 million€) to one or more brain researchers who have had a ground-breaking impact on brain research.
When X-linked genes evade silencing on the “inactive” chromosome in XX cells, some protect women from diseases such as cancer, but others seem to promote conditions such as autoimmunity.
Gene or genome editing describes a process by which targeted and deliberate changes to the DNA sequence are made. Recent advances in programmable nucleases, including the CRISPR-Cas9 system, mean that manipulating DNA has never been easier, which could fundamentally alter the way we treat and ...
Making changes to the molecular messengers that create proteins might offer flexible therapies for cancer, pain or high cholesterol, in addition to genetic disorders.
Austrian pediatrician Andreas Rett first recognized the syndrome that would later bear his name in the mid-1960s. The first description in English, published in 1983, detailed a “progressive syndrome of autism” and other traits in 35 girls.