8legs: Petah tikva, israel

Your Followed Topics

#1 out of 2

health11h ago

Eight-month-old patient with WOREE syndrome received brain-delivery gene replacement therapy.
Therapy aimed to restore WWOX gene function to treat WOREE syndrome.
The drug delivery was via a delicate neurosurgical procedure delivering a functioning gene copy into the brain.
International collaboration and regulatory approvals supported the treatment.
Preclinical data indicated gene replacement restored expression and improved outcomes in models.
The child was discharged with no recurring seizures after treatment.
Researchers emphasized this as a potential first-of-its-kind option for rare genetic diseases.
WOREE is extremely rare, with only 60 to 90 cases identified worldwide.
The gene therapy was licensed to a US biotech company, Mahzi Therapeutics.
Experts hope this approach could lead to new treatments for similar genetic disorders.

Vote 0

#2 out of 2100.00%

An eight‑month‑old with WOREE syndrome received an experimental WWOX gene therapy directly in the brain at Schneider Children’s Medical Center.
The treatment was delivered at Schneider Children’s Medical Center of Israel in Petah Tikva as part of a compassionate‑use program.
Researchers used an AAV9 vector to deliver a healthy copy of the WWOX gene to neurons, with preclinical studies supporting the concept.
Mahzi Therapeutics is advancing the program toward clinical application and coordinating translational and regulatory activities.
WWOX‑related epileptic encephalopathy causes early‑onset, drug‑resistant seizures and severe developmental impairment.
Early follow-up showed no recurrence of severe seizures during the initial observation period after treatment.
The program builds on HUJI research linking WWOX to nervous system development and function.
The initial success is described as a milestone toward personalized therapies for rare genetic epilepsies.
The infant's condition originated from a genetic defect common in Yemeni Jewish ancestry but with variants worldwide.
The therapy represents a collaboration among Israeli and US scientists and regulatory partners.

Vote 0

Unlimited Access

Personalized Feed

Full Experience

By continuing, you agree to the Privacy Policy.. You also agree to receive our newsletters, you can opt-out any time.

Create an account and enjoy content that interests you with your personalized feed

Unlimited Access

Personalized Feed

Full Experience

By continuing, you agree to the Privacy Policy.. You also agree to receive our newsletters, you can opt-out any time.