Rett In The News

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Two unpublished studies detail improved methods for delivering gene therapies to the brain: One involves a type of stem cell that can produce gene-altering proteins on-site;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.

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.

Problems with the formation of DNA-filled droplets that control gene expression may contribute to Rett syndrome, a new study suggests.

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 ...

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.

OHSU study first to use programmable RNA editing to repair gene that contributes to Rett syndrome.

Our executive director, Monica Coenraads, and her daughter are featured on the front page of the NYT science section.

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.

There’s more than meets the eye — here are some tips to help avoid confusion.

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.