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Refinado por: assunto: Genes remover assunto: Genetic Vectors remover
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1
Nonviral Vectors for Gene Therapy
Nonviral Vectors for Gene Therapy
Nonviral Vectors for Gene Therapy
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Nonviral Vectors for Gene Therapy

Mien-Chie Huang, Leaf Wagner, Ernst Hung Leaf Huang; Mien-chie Hung; Ernst Wagner Ernst Wagner Leaf Huang

Burlington Academic Press 1999

Acesso online. A biblioteca também possui exemplares impressos.

2
Treatment of a metabolic liver disease by in vivo genome base editing in adult mice
Treatment of a metabolic liver disease by in vivo genome base editing in adult mice
Material Type:
Artigo 		Adicionar ao Meu Espaço

Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

Villiger, Lukas ; Grisch-Chan, Hiu Man ; Lindsay, Helen ; Ringnalda, Femke ; Pogliano, Chiara B ; Allegri, Gabriella ; Fingerhut, Ralph ; Häberle, Johannes ; Matos, Joao ; Robinson, Mark D ; Thöny, Beat ; Schwank, Gerald

Nature medicine, 2018-10, Vol.24 (10), p.1519-1525 [Periódico revisado por pares]

United States: Nature Publishing Group

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3
Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial
Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial
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Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial

Ghazi, Nicola G. ; Abboud, Emad B. ; Nowilaty, Sawsan R. ; Alkuraya, Hisham ; Alhommadi, Abdulrahman ; Cai, Huimin ; Hou, Rui ; Deng, Wen-Tao ; Boye, Sanford L. ; Almaghamsi, Abdulrahman ; Al Saikhan, Fahad ; Al-Dhibi, Hassan ; Birch, David ; Chung, Christopher ; Colak, Dilek ; LaVail, Matthew M. ; Vollrath, Douglas ; Erger, Kirsten ; Wang, Wenqiu ; Conlon, Thomas ; Zhang, Kang ; Hauswirth, William ; Alkuraya, Fowzan S.

Human genetics, 2016-03, Vol.135 (3), p.327-343 [Periódico revisado por pares]

Berlin/Heidelberg: Springer Berlin Heidelberg

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4
An essential receptor for adeno-associated virus infection
An essential receptor for adeno-associated virus infection
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An essential receptor for adeno-associated virus infection

Pillay, S ; Meyer, N L ; Puschnik, A S ; Davulcu, O ; Diep, J ; Ishikawa, Y ; Jae, L T ; Wosen, J E ; Nagamine, C M ; Chapman, M S ; Carette, J E

Nature (London), 2016-02, Vol.530 (7588), p.108-112 [Periódico revisado por pares]

England: Nature Publishing Group

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5
Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector
Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector
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Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector

Cideciyan, Artur V. ; Sudharsan, Raghavi ; Dufour, Valérie L. ; Massengill, Michael T. ; Iwabe, Simone ; Swider, Malgorzata ; Lisi, Brianna ; Sumaroka, Alexander ; Marinho, Luis Felipe ; Appelbaum, Tatyana ; Rossmiller, Brian ; Hauswirth, William W. ; Jacobson, Samuel G. ; Lewin, Alfred S. ; Aguirre, Gustavo D. ; Beltran, William A.

Proceedings of the National Academy of Sciences - PNAS, 2018-09, Vol.115 (36), p.E8547-E8556 [Periódico revisado por pares]

United States: National Academy of Sciences

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6
Evolving AAV-delivered therapeutics towards ultimate cures
Evolving AAV-delivered therapeutics towards ultimate cures
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Evolving AAV-delivered therapeutics towards ultimate cures

He, Xiangjun ; Urip, Brian Anugerah ; Zhang, Zhenjie ; Ngan, Chun Christopher ; Feng, Bo

Journal of molecular medicine (Berlin, Germany), 2021-05, Vol.99 (5), p.593-617 [Periódico revisado por pares]

Berlin/Heidelberg: Springer Berlin Heidelberg

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7
A dual‐AAV approach restores fast exocytosis and partially rescues auditory function in deaf otoferlin knock‐out mice
A dual‐AAV approach restores fast exocytosis and partially rescues auditory function in deaf otoferlin knock‐out mice
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A dual‐AAV approach restores fast exocytosis and partially rescues auditory function in deaf otoferlin knock‐out mice

Al‐Moyed, Hanan ; Cepeda, Andreia P ; Jung, SangYong ; Moser, Tobias ; Kügler, Sebastian ; Reisinger, Ellen

EMBO molecular medicine, 2019-01, Vol.11 (1), p.n/a [Periódico revisado por pares]

England: EMBO Press

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8
Biomaterial-guided delivery of gene vectors for targeted articular cartilage repair
Biomaterial-guided delivery of gene vectors for targeted articular cartilage repair
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Biomaterial-guided delivery of gene vectors for targeted articular cartilage repair

Cucchiarini, Magali ; Madry, Henning

Nature reviews. Rheumatology, 2019-01, Vol.15 (1), p.18-29 [Periódico revisado por pares]

United States: Nature Publishing Group

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9
Cancer therapy with a CRISPR-assisted telomerase-activating gene expression system
Cancer therapy with a CRISPR-assisted telomerase-activating gene expression system
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Cancer therapy with a CRISPR-assisted telomerase-activating gene expression system

Dai, Wei ; Xu, Xinhui ; Wang, Danyang ; Wu, Jian ; Wang, Jinke

Oncogene, 2019-05, Vol.38 (21), p.4110-4124 [Periódico revisado por pares]

England: Nature Publishing Group

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10
Astrocyte-selective AAV gene therapy through the endogenous GFAP promoter results in robust transduction in the rat spinal cord following injury
Astrocyte-selective AAV gene therapy through the endogenous GFAP promoter results in robust transduction in the rat spinal cord following injury
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Astrocyte-selective AAV gene therapy through the endogenous GFAP promoter results in robust transduction in the rat spinal cord following injury

Griffin, Jarred M ; Fackelmeier, Barbara ; Fong, Dahna M ; Mouravlev, Alexander ; Young, Deborah ; O'Carroll, Simon J

Gene therapy, 2019-05, Vol.26 (5), p.198-210 [Periódico revisado por pares]

England: Nature Publishing Group

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Deste Autor:

  1. Hung, M
  2. Wagner, E
  3. Ernst Wagner
  4. Huang, L
  5. Leaf Huang

Neste Assunto:

  1. Genes
  2. Genetic Vectors
  3. Genetic Vectors
  4. Genes
  5. Science & Technology

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