Muscle Gene Therapy
暫譯: 肌肉基因療法
Duan, Dongsheng, Mendell, Jerry R.
- 出版商: Springer
- 出版日期: 2019-04-11
- 售價: $12,900
- 貴賓價: 9.5 折 $12,255
- 語言: 英文
- 頁數: 811
- 裝訂: Hardcover - also called cloth, retail trade, or trade
- ISBN: 3030030946
- ISBN-13: 9783030030940
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商品描述
Muscle disease represents an important health threat to the general population. Unlike diseases affecting other tissues/organs, therapeutic options are quite limited for many inherited muscle diseases such as Duchenne muscular dystrophy. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. A variety of new strategies, such as exon-skipping and RNAi, have greatly expanded the scope of muscle gene therapy beyond the traditional gene replacement approach. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy.
商品描述(中文翻譯)
肌肉疾病對一般人群構成了重要的健康威脅。與影響其他組織/器官的疾病不同，許多遺傳性肌肉疾病（如杜氏肌肉萎縮症）的治療選擇相當有限。基本上沒有治癒的方法。基因療法在糾正遺傳缺陷和最終實現這些疾病的完全康復方面具有很大的潛力。在過去幾年中，肌肉基因療法領域取得了顯著進展。各種新策略，如外顯子跳過（exon-skipping）和RNA干擾（RNAi），大大擴展了肌肉基因療法的範疇，超越了傳統的基因替代方法。新型基因傳遞載體的開發顯著提高了肌肉基因傳遞的特異性和效率。對病毒載體免疫反應的新知識為克服免疫障礙提供了新的見解。最重要的是，該領域終於從小型實驗動物模型轉向人類患者。本書將匯聚肌肉基因轉移領域的領導者，提供有關肌肉基因療法進展的最新概述，並強調肌肉基因療法的重要臨床應用。
作者簡介
Dongsheng Duan, Ph.D., is the Margaret Proctor Mulligan Professor in Medical Research, at the Department of Molecular Microbiology & Immunology, Neurology, Bioengineering, and Biomedical Sciences at the University of Missouri. He received his medical degree from the West China University of Medical Science, Chengdu, China in 1987 and his Ph.D. degree from the University of Pennsylvania, Philadelphia, USA in 1997. His research in last two decades has focused on the development of adeno-associated virus as a gene therapy vector, pathogenic mechanisms of Duchenne muscular dystrophy, dystrophin biology and preclinical gene therapy for Duchenne muscular dystrophy in murine and canine models.
Jerry R Mendell, M.D. is the Curran-Peters Research Chair and Professor of Pediatrics and Neurology. His career has been devoted to translational clinical science, beginning as a post doctoral fellow at the National Institutes of Health. His life-long focus has been to make a difference in lives burdened with untreatable neuromuscular disease. He has contributed significantly to the muscular dystrophies and spinal muscular atrophy, at first using pharmaceutical tools and since 1999, the time of his first gene therapy trial, he has been dedicated to the successful replacement of mutant genes that undermine the lives of innocent victims.
作者簡介(中文翻譯)
董勝端博士是密蘇里大學分子微生物學與免疫學、神經學、生物工程及生物醫學科學系的瑪格麗特·普羅克特·穆利根醫學研究教授。他於1987年在中國成都的華西醫科大學獲得醫學學位，並於1997年在美國費城的賓夕法尼亞大學獲得博士學位。在過去的二十年中，他的研究專注於開發腺相關病毒作為基因治療載體、杜氏肌營養不良症的致病機制、肌動蛋白生物學以及在小鼠和犬類模型中進行的杜氏肌營養不良症的臨床前基因治療。
傑瑞·R·門德爾醫生是庫蘭-彼得斯研究主席及小兒科和神經學教授。他的職業生涯致力於轉化臨床科學，最初作為國立衛生研究院的博士後研究員。他一生的重點是改善那些受到無法治療的神經肌肉疾病困擾的生命。他對肌肉萎縮症和脊髓性肌萎縮症做出了重要貢獻，最初使用藥物工具，自1999年首次基因治療試驗以來，他一直致力於成功替換那些破壞無辜受害者生活的突變基因。
