Titlebook: Gene Delivery and Therapy for Neurological Disorders; Xuenong Bo,Joost Verhaagen Book 2015 Springer Science+Business Media New York 2015 A

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AAV Gene Therapy Strategies for Lysosomal Storage Disorders with Central Nervous System InvolvementLSDs that have central nervous system (CNS) involvement, where enzyme replacement therapy (ERT), the standard of care for LSDs, is ineffective in treating the neurological features of these diseases. Recombinant adeno-associated virus (AAV) vectors have emerged as the most efficient and promising ge

减至最低

Gene Therapy in Spinal Muscular Atrophy (SMA) Models Using Intracerebroventricular Injection into Ndels of the disease. Preclinical animal studies often proceed to larger animal models and are subsequently incorporated into the final design of the clinical trials. Recent gene therapy advancements with preclinical animal models of spinal muscular atrophy (SMA) have made the move from the bench res

COUCH

Gene Therapy for Chronic Pain: How to Manipulate and Unravel Pain Control Circuits from the Brain?,uring the last decades, the available analgesic drugs remained considerably unchanged. Gene therapy emerged as an important tool in the pain field. Studies in experimental pain models consisted on blockade of nociceptive transmission at the spinal cord by peripheral delivery of viral vectors (mainly

GEST

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0893-2336 results.Contains key notes and implementation advice from th.This volume aims to explore the latest developments in adeno-associated viral and lentiviral vectors as well as the gene therapy strategies for the most common neurological disorders, followed by chapters that include step-by-step guides t

Awning

Acute diarrhea in the Dominican Republicdies have demonstrated the efficiency of these vectors both at preclinical and clinical development stages. The current chapter will describe the basic features of AAV vectors, list few examples of their applications as a therapeutic tool to treat diseases of the central nervous system, and discuss progress in the manufacturing process.

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失望未来

Adeno-Associated Vectors for Gene Delivery to the Nervous System,dies have demonstrated the efficiency of these vectors both at preclinical and clinical development stages. The current chapter will describe the basic features of AAV vectors, list few examples of their applications as a therapeutic tool to treat diseases of the central nervous system, and discuss progress in the manufacturing process.

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Gene Therapy for Epilepsies,ndidates for epilepsy gene therapy. The techniques described here include adeno-associated viral vector production, genomic titering of the vector, stereotaxic neurosurgery for intracerebral vector administration, and the kainic acid seizure model.

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Book 2015ost common neurological disorders, followed by chapters that include step-by-step guides to viral vector-based gene delivery in animal models used in the authors’ laboratories. Although safe gene manipulation in neural cells can be achieved, it may still be years away from efficacious gene-based tre