Fidest – Agenzia giornalistica/press agency

Quotidiano di informazione – Anno 31 n°159

Two Rare and Devastating Neurodegenerative Disorders

Posted by fidest press agency su domenica, 10 febbraio 2019

Neurogene, Inc., a company with a mission to bring life-changing medicines to patients and families affected by rare neurological diseases, today announced that researchers presented preliminary, unpublished “proof of concept” data in mice evaluating the safety and efficacy of adeno-associated virus (AAV) gene therapy for AGU and CMT4J, two rare neurological diseases, at the 15th Annual WORLDSymposiumTM conference in Orlando, Florida.
The potential therapies are being investigated under a collaboration agreement between Neurogene and UT Southwestern Medical Center through multiple sponsored agreements. Neurogene provides financial support for these and other related research projects, including pre-clinical studies for AGU and CMT4J.AAV gene therapy is being investigated as a potential treatment for aspartylglucosaminuria (AGU), a neurodegenerative disease caused by dysfunction of the AGA gene. The unpublished data, which have not been peer reviewed, consider the safety and efficacy of gene therapy for these diseases in mice in support of potential future clinical trials. Xin Chen, Ph.D., Instructor of Pediatrics at UT Southwestern Medical Center, presented data evaluating the effects in mice lacking a functional AGA gene, using intravenous or intrathecal administration with low and high doses, and data were reported for early symptomatic mice. Dr. Chen’s team reported data indicating AGU gene therapy resulted in dose-dependent, complete or near-complete elimination of toxic substrate in central and peripheral tissues and body fluids and that treatment was well-tolerated at supraphysiological levels.

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