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Neurologic orphan diseases:Emerging innovations and role for genetic treatments

World Journal of Experimental Medicine 2023年第4期13卷 59-74页

作者： ivelina p.kioutchoukova Devon T Foster Rajvi N Thakkar Marco A Foreman Brandon J Burgess Rebecca M Toms Eduardo E Molina Valero Brandon Lucke-Wold College of Medicine University of FloridaGainesvilleFL 32611United States Florida International University Herbert Wertheim College of Medicine Florida International University Herbert Wertheim College of MedicineMiamiFL 33199United States Department of Neurosurgery University of FloridaGainesvilleFL 32611United States

Orphan diseases are rare diseases that affect less than 200000 individuals within the United *** orphan diseases are of neurologic and genetic *** the current advances in technology,more funding has been devoted to developing therapeutic agents for patients with these *** our review,we highlight emerging options for patients with neurologic orphan diseases,specifically including diseases resulting in muscular deterioration,epilepsy,seizures,neurodegenerative movement disorders,inhibited cognitive development,neuron deterioration,and *** extensive literature review,gene therapy offers a promising route for the treatment of neurologic orphan *** use of clustered regularly interspaced palindromic repeats/Cas9 has demonstrated positive results in experiments investigating its role in several ***,the use of adeno-associated viral vectors has shown improvement in survival,motor function,and developmental milestones,while also demonstrating reversal of sensory ataxia and cardiomyopathy in Friedreich ataxia *** oligonucleotides have also been used in some neurologic orphan diseases with positive *** target of rapamycin inhibitors are currently being investigated and have reduced abnormal cell growth,proliferation,and *** innovations and the role of genetic treatments open a new window of opportunity for the treatment of neurologic orphan diseases.

关键词： Neurologic orphan diseases Gene therapy Clustered regularly interspaced palindromic repeats/Cas9 Antisense oligonucleotides Adeno-associated virus mTOR inhibitors

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