Birth of a New Therapeutic Platform: 47 Years of Adeno-associated Virus Biology From Virus Discovery to Licensed Gene Therapy

被引：16
作者：
Flotte, Terence R. [1 ]
机构：
[1] Univ Massachusetts, Sch Med, Dept Pediat, Gene Therapy Ctr, Worcester, MA USA
来源：
MOLECULAR THERAPY | 2013年 / 21卷 / 11期
关键词：
ADENO-ASSOCIATED VIRUS; LEBER CONGENITAL AMAUROSIS; PHASE-I TRIAL; TRANSMEMBRANE CONDUCTANCE REGULATOR; CYSTIC-FIBROSIS; MESSENGER-RNA; CLINICAL-TRIAL; WILD-TYPE; INTRAMUSCULAR INJECTION; RECOMBINANT PLASMID;
D O I：
10.1038/mt.2013.226
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：
引用
收藏
页码：1976 / 1981
页数：6
相关论文
共 99 条
[81]   Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors [J].
Snyder, RO ;
Miao, CH ;
Patijn, GA ;
Spratt, SK ;
Danos, O ;
Nagy, D ;
Gown, AM ;
Winther, B ;
Meuse, L ;
Cohen, LK ;
Thompson, AR ;
Kay, MA .
NATURE GENETICS, 1997, 16 (03) :270-276
[82]   INVITRO RESOLUTION OF COVALENTLY JOINED AAV CHROMOSOME ENDS [J].
SNYDER, RO ;
SAMULSKI, RJ ;
MUZYCZKA, N .
CELL, 1990, 60 (01) :105-113
[83]   FEATURES OF THE ADENOASSOCIATED VIRUS ORIGIN INVOLVED IN SUBSTRATE RECOGNITION BY THE VIRAL REP PROTEIN [J].
SNYDER, RO ;
IM, DS ;
NI, TH ;
XIAO, X ;
SAMULSKI, RJ ;
MUZYCZKA, N .
JOURNAL OF VIROLOGY, 1993, 67 (10) :6096-6104
[84]   EVIDENCE FOR COVALENT ATTACHMENT OF THE ADENOASSOCIATED VIRUS (AAV) REP PROTEIN TO THE ENDS OF THE AAV GENOME [J].
SNYDER, RO ;
IM, DS ;
MUZYCZKA, N .
JOURNAL OF VIROLOGY, 1990, 64 (12) :6204-6213
[85]   Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors [J].
Snyder, RO ;
Miao, C ;
Meuse, L ;
Tubb, J ;
Donahue, BA ;
Lin, HF ;
Stafford, DW ;
Patel, S ;
Thompson, AR ;
Nichols, T ;
Read, MS ;
Bellinger, DA ;
Brinkhous, KM ;
Kay, MA .
NATURE MEDICINE, 1999, 5 (01) :64-70
[86]   NUCLEOTIDE-SEQUENCE AND ORGANIZATION OF THE ADENO-ASSOCIATED VIRUS-2 GENOME [J].
SRIVASTAVA, A ;
LUSBY, EW ;
BERNS, KI .
JOURNAL OF VIROLOGY, 1983, 45 (02) :555-564
[87]   Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2 [J].
Testa, Francesco ;
Maguire, Albert M. ;
Rossi, Settimio ;
Pierce, Eric A. ;
Melillo, Paolo ;
Marshall, Kathleen ;
Banfi, Sandro ;
Surace, Enrico M. ;
Sun, Junwei ;
Acerra, Carmela ;
Wright, J. Fraser ;
Wellman, Jennifer ;
High, Katherine A. ;
Auricchio, Alberto ;
Bennett, Jean ;
Simonelli, Francesca .
OPHTHALMOLOGY, 2013, 120 (06) :1283-1291
[88]   A HUMAN PARVOVIRUS, ADENO-ASSOCIATED VIRUS, AS A EUKARYOTIC VECTOR - TRANSIENT EXPRESSION AND ENCAPSIDATION OF THE PROCARYOTIC GENE FOR CHLORAMPHENICOL ACETYLTRANSFERASE [J].
TRATSCHIN, JD ;
WEST, MHP ;
SANDBANK, T ;
CARTER, BJ .
MOLECULAR AND CELLULAR BIOLOGY, 1984, 4 (10) :2072-2081
[89]   NEGATIVE AND POSITIVE REGULATION IN TRANS OF GENE-EXPRESSION FROM ADENOASSOCIATED VIRUS VECTORS IN MAMMALIAN-CELLS BY A VIRAL REP GENE-PRODUCT [J].
TRATSCHIN, JD ;
TAL, J ;
CARTER, BJ .
MOLECULAR AND CELLULAR BIOLOGY, 1986, 6 (08) :2884-2894
[90]   ALTERNATE MESSENGER-RNA SPLICING IS REQUIRED FOR SYNTHESIS OF ADENO-ASSOCIATED VIRUS VP1 CAPSID PROTEIN [J].
TREMPE, JP ;
CARTER, BJ .
JOURNAL OF VIROLOGY, 1988, 62 (09) :3356-3363
12345678910