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Gene Therapy Reverses Symptoms of Parkinson's Disease

A gene therapy called NLX-P101 dramatically reduces movement impairment in Parkinson's patients, according to results of a Phase 2 study published today in the journal Lancet Neurology. The approach introduces a gene into the brain to normalize chemical signaling.

The study is the first successful randomized, double-blind clinical trial of a gene therapy for Parkinson's or any neurologic disorder, and it represents the culmination of 20 years of research by study co-authors Dr. Michael Kaplitt, vice chairman for research in the Department of Neurological Surgery at Weill Cornell Medical College and a neurosurgeon at NewYork-Presbyterian Hospital/Weill Cornell Medical Center, and Dr. Matthew During, originally at Yale University and now professor of molecular virology, immunology and medical genetics, neuroscience and neurological surgery at the Ohio State University.

"Patients who received NLX-P101 showed a significant reduction in the motor symptoms of Parkinson's, including tremor, rigidity and difficulty initiating movement," says Dr. Kaplitt, who pioneered the approach and helped design the clinical trial. "This not only confirms the results of our Phase 1 trial performed at NewYork-Presbyterian/Weill Cornell but also represents a major milestone in the development of gene therapy for a wide range of neurological diseases."

"This is great news for the 1.5 million Americans living with Parkinson's disease," adds Dr. During, who is the co-inventor, with Dr. Kaplitt, of the gene therapy procedure. "Since this is also the first gene therapy study for a neurological disease to achieve success in a rigorous randomized, double-blind design compared with a sham group, this is also a crucial step forward toward finally bringing gene therapy into clinical practice for patients with debilitating brain disorders."

Gene Therapy:遺伝子治療 Symptoms:症状 Parkinson's Disease:パーキンソン病
movement impairment:運動障害 according to:~によると Phase 2 study:第2相試験 Neurology:神経学(Lancet Neurologyは医学雑誌の名称) brain:脳 normalize:正常化する signaling:シグナル伝達
randomized:無作為化 double-blind clinical trial:二重盲検臨床試験 neurologic disorder:神経障害 culmination:成就,集大成 co-authors:共著者 vice chairman:副学科長 Neurological Surgery:脳神経外科 neurosurgeon:脳神経外科医 molecular virology:分子ウイルス学 immunology:免疫学 medical genetics:遺伝医学 neuroscience:神経科学
motor symptoms:運動症状 tremor:振戦 rigidity:硬直 initiating:始めること represents a major milestone:画期的なことである a wide range of:幅広い neurological diseases:神経疾患
co-inventor:共同発明者 achieve success:成功を収める rigorous:厳密な sham group:偽処置群 a step forward:一歩前進 debilitating brain disorders:消耗性脳疾患



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