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Human gene therapy(6,603)
ISSN: 1043-0342
1    
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:29  Issue:3  Page:285-298  

Hinderer Christian;   Katz Nathan;   Buza Elizabeth L;   Dyer Cecilia;   Goode Tamara;   ...

来源数据库:[MEDLINE, Scopus]        

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2    
Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:26  Issue:7  Page:432-442  

Wang Dan;   Mou Haiwei;   Li Shaoyong;   Li Yingxiang;   Hough Soren;   ...

来源数据库:[Web of Science, Scopus]         被引频次:119

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3    
Treatment of Leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: Short-term results of a phase I trial 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:19  Issue:10  Page:979-990  

Hauswirth William W;   Aleman Tomas S;   Kaushal Shalesh;   Cideciyan Artur V;   Schwartz Sharon B;   ...

来源数据库:[Web of Science, Scopus]         被引频次:605

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4    
Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: Implications for gene therapy ... 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:21  Issue:6  Page:704-712  

Boutin Sylvie;   Monteilhet Virginie;   Veron Philippe;   Leborgne Christian;   Benveniste Olivier;   ...

来源数据库:[Web of Science, Scopus]         被引频次:335

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5    
Oligonucleotide Therapies: The Past and the Present 期刊论文

Human Gene Therapy   ISSN:1043-0342,1557-7422  Volume:26  Issue:8  Page:475-485  

Lundin Karin E;   Gissberg Olof;   Smith C.I. Edvard;  

来源数据库:[Web of Science, Scopus]         被引频次:97

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6    
Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human SGSH and SUMF1 cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease:... 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:25  Issue:6  Page:56-516  

Tardieu Marc;   Zérah Michel;   Husson Béatrice;   de Bournonville Stéphanie;   Deiva Kumaran;   ...

来源数据库:[Web of Science, Scopus]         被引频次:115

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7    
Recognition of Glioma Stem Cells by Genetically Modified T Cells Targeting EGFRvIII and Development of Adoptive Cell Therapy for Glioma 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:23  Issue:10  Page:143-1053  

Morgan Richard A;   Johnson Laura A;   Davis Jeremy L;   Zheng Zhili;   Woolard Kevin D;   ...

来源数据库:[Web of Science, Scopus]         被引频次:147

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8    
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:27  Issue:7  Page:478-496  

Hocquemiller Michaël;   Giersch Laura;   Audrain Mickael;   Parker Samantha;   Cartier Nathalie;  

来源数据库:[Web of Science, Scopus]         被引频次:73

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9    
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α 1-antitrypsin: Interim results 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:22  Issue:10  Page:1239-1247  

Flotte Terence R;   Trapnell Bruce C;   Humphries Margaret;   Carey Brenna;   Calcedo Roberto;   ...

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10    
Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing alpha(1)-Antitrypsin: Interim Results 期刊论文

HUMAN GENE THERAPY     ISSN:1043-0342  Volume:22  Issue:10  Page:1239-1247  

Flotte TR;   Trapnell BC;   Humphries M;   Carey B;   Calcedo R;   ...

被引频次:171

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11    
The regulation of differentiation in mesenchymal stem cells 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:21  Issue:10  Page:1226-1238  

Augello Andrea;   De Bari Cosimo;  

来源数据库:[Web of Science, Scopus]         被引频次:192

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12    
Immunological aspects of allogeneic mesenchymal stem cell therapies 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:21  Issue:12  Page:1641-1655  

Griffin Matthew D;   Ritter Thomas;   Mahon Bernard P;  

来源数据库:[Web of Science, Scopus]         被引频次:181

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13    
Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:20  Issue:9  Page:999-1004  

Cideciyan Artur V;   Hauswirth William W;   Aleman Tomas S;   Kaushal Shalesh;   Schwartz Sharon B;   ...

来源数据库:[Web of Science, Scopus]         被引频次:227

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14    
Delivery and Specificity of CRISPR/Cas9 Genome Editing Technologies for Human Gene Therapy 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:26  Issue:7  Page:443-451  

Gori Jennifer L;   Hsu Patrick D;   Maeder Morgan L;   Shen Shen;   Welstead G. Grant;   ...

来源数据库:[Web of Science, Scopus]         被引频次:74

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15    
Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-Globin Gene 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:27  Issue:2  Page:148-165  

Negre Olivier;   Eggimann Anne-Virginie;   Beuzard Yves;   Ribeil Jean-Antoine;   Bourget Philippe;   ...

来源数据库:[Web of Science, Scopus]         被引频次:60

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16    
Innate Immunity to Adenovirus 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:25  Issue:4  Page:265-284  

Hendrickx Rodinde;   Stichling Nicole;   Koelen Jorien;   Kuryk Lukasz;   Lipiec Agnieszka;   ...

来源数据库:[Web of Science, Scopus]         被引频次:80

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17    
Bone marrow mesenchymal stem cells: Historical overview and concepts 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:21  Issue:9  Page:1045-1056  

Charbord Pierre;  

来源数据库:[Web of Science, Scopus]         被引频次:163

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18    
Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:23  Issue:4  Page:382-389  

Samaranch Lluis;   Salegio Ernesto A;   San Sebastian Waldy;   Kells Adrian P;   Foust Kevin D;   ...

来源数据库:[Web of Science, Scopus]         被引频次:127

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19    
Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success—A Personal Perspective 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:26  Issue:5  Page:257-265  

Hastie Eric;   Samulski R. Jude;  

来源数据库:[Web of Science, Scopus]         被引频次:67

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20    
Challenges in CRISPR/CAS9 Delivery: Potential Roles of Nonviral Vectors 期刊论文

Human Gene Therapy     ISSN:1043-0342  Volume:26  Issue:7  Page:452-462  

Li Ling;   He Zhi-Yao;   Wei Xia-Wei;   Gao Guang-Ping;   Wei Yu-Quan;  

来源数据库:[Web of Science, Scopus]         被引频次:63

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