Current Clinical Applications of In Vivo Gene Therapy with AAVs

被引:489
作者
Mendell, Jerry R. [1 ,2 ]
Al-Zaidy, Samiah A. [3 ]
Rodino-Klapac, Louise R. [4 ]
Goodspeed, Kimberly [5 ]
Gray, Steven J. [5 ]
Kay, Christine N. [6 ]
Boye, Sanford L. [7 ]
Boye, Shannon E. [8 ]
George, Lindsey A. [9 ,10 ,11 ]
Salabarria, Stephanie [12 ]
Corti, Manuela [13 ,14 ]
Byrne, Barry J. [13 ,14 ]
Tremblay, Jacques P. [15 ]
机构
[1] Nationwide Childrens Hosp, Ctr Gene Therapy, Abigail Wexner Res Inst, Columbus, OH USA
[2] Ohio State Univ, Dept Pediat & Neurol, Columbus, OH 43210 USA
[3] Al Zaidy & Associates LLC, Columbus, OH USA
[4] Sarepta Therapeut Inc, Cambridge, MA USA
[5] UT Southwestern Med Ctr, Dept Pediat, Powell Gene Therapy Ctr, Dallas, TX USA
[6] Vitreoretinal Associates, Gainesville, FL USA
[7] Univ Florida, Dept Pediat, Powell Gene Therapy Ctr, Gainesville, FL USA
[8] Univ Florida, Div Cellular & Mol Therapeut, Gainesville, FL USA
[9] Div Hematol, Philadelphia, PA USA
[10] Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA USA
[11] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
[12] Univ Penn, Dept Pediat, Perelman Sch Med, Philadelphia, PA USA
[13] Univ Florida, Coll Med, Dept Pediat, Gainesville, FL USA
[14] Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL USA
[15] CHUQ Univ Laval, Ctr Rech, Quebec City, PQ, Canada
基金
美国国家卫生研究院;
关键词
D O I
10.1016/j.ymthe.2020.12.007
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases affect over 30 million Americans. For more than 30 years, hundreds of researchers have maintained that genetic modifications would provide effective treatments for many inherited human diseases, offering durable and possibly curative clinical benefit with a single treatment. This review is limited to gene therapy using adeno-associated virus (AAV) because the gene delivered by this vector does not integrate into the patient genome and has a low immunogenicity. There are now five treatments approved for commercialization and currently available, i.e., Luxturna, Zolgensma, the two chimeric antigen receptor T cell (CAR-T) therapies (Yescarta and Kymriah), and Strimvelis (the gammaretrovirus approved for adenosine deaminase-severe combined immunodeficiency [ADA-SCID] in Europe). Dozens of other treatments are under clinical trials. The review article presents a broad overview of the field of therapy by in vivo gene transfer. We review gene therapy for neuromuscular disorders (spinal muscular atrophy [SMA]; Duchenne muscular dystrophy [DMD]; X-linked myotubular myopathy [XLMTM]; and diseases of the central nervous system, including Alzheimer's disease, Parkinson?s disease, Canavan disease, aromatic L-amino acid decarboxylase [AADC] deficiency, and giant axonal neuropathy), ocular disorders (Leber congenital amaurosis, age-related macular degeneration [AMD], choroideremia, achromatopsia, retinitis pigmentosa, and X-linked retinoschisis), the bleeding disorder hemophilia, and lysosomal storage disorders.
引用
收藏
页码:464 / 488
页数:25
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