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Refinado por: Nome da Publicação: Gene Therapy remover assunto: Gene Therapy remover
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1
Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy
Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy
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Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy

Nie, Jiali ; Han, Yu ; Jin, Zhiyuan ; Hang, Weijian ; Shu, Hongyang ; Wen, Zheng ; Ni, Li ; Wang, Dao Wen

Gene therapy, 2023-06, Vol.30 (6), p.520-527 [Periódico revisado por pares]

England: Nature Publishing Group

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2
Retroviral gene therapy in Germany with a view on previous experience and future perspectives
Retroviral gene therapy in Germany with a view on previous experience and future perspectives
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Retroviral gene therapy in Germany with a view on previous experience and future perspectives

Morgan, Michael A ; Galla, Melanie ; Grez, Manuel ; Fehse, Boris ; Schambach, Axel

Gene therapy, 2021-09, Vol.28 (9), p.494-512 [Periódico revisado por pares]

England: Nature Publishing Group

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3
Targeted gene therapy into a safe harbor site in human hematopoietic progenitor cells
Targeted gene therapy into a safe harbor site in human hematopoietic progenitor cells
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Targeted gene therapy into a safe harbor site in human hematopoietic progenitor cells

Rodriguez-Fornes, Fatima ; Quintana-Bustamante, Oscar ; Lozano, M Luz ; Segovia, Jose C ; Bueren, Juan A ; Guenechea, Guillermo

Gene therapy, 2020-09, Vol.27 (9), p.435-450 [Periódico revisado por pares]

England: Nature Publishing Group

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4
Treating osteoarthritis via gene therapy with rejuvenation factors
Treating osteoarthritis via gene therapy with rejuvenation factors
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Treating osteoarthritis via gene therapy with rejuvenation factors

Zhang, Jiahe ; Liu, Guang-Hui ; Qu, Jing ; Song, Moshi

Gene therapy, 2020-08, Vol.27 (7-8), p.309-311 [Periódico revisado por pares]

England: Nature Publishing Group

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5
Lentiviral gene therapy and vitamin B3 treatment enable granulocytic differentiation of G6PC3-deficient induced pluripotent stem cells
Lentiviral gene therapy and vitamin B3 treatment enable granulocytic differentiation of G6PC3-deficient induced pluripotent stem cells
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Lentiviral gene therapy and vitamin B3 treatment enable granulocytic differentiation of G6PC3-deficient induced pluripotent stem cells

Hoffmann, Dirk ; Kuehle, Johannes ; Lenz, Daniela ; Philipp, Friederike ; Zychlinski, Daniela ; Lachmann, Nico ; Moritz, Thomas ; Steinemann, Doris ; Morgan, Michael ; Skokowa, Julia ; Klein, Christoph ; Schambach, Axel

Gene therapy, 2020-06, Vol.27 (6), p.297-306 [Periódico revisado por pares]

England: Nature Publishing Group

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6
Gene therapy of hematological disorders: current challenges
Gene therapy of hematological disorders: current challenges
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Gene therapy of hematological disorders: current challenges

Al-Saif, Amr M

Gene therapy, 2019-08, Vol.26 (7-8), p.296-307 [Periódico revisado por pares]

England: Nature Publishing Group

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7
In situ regeneration of retinal pigment epithelium by gene transfer of E2F2: a potential strategy for treatment of macular degenerations
In situ regeneration of retinal pigment epithelium by gene transfer of E2F2: a potential strategy for treatment of macular degenerations
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In situ regeneration of retinal pigment epithelium by gene transfer of E2F2: a potential strategy for treatment of macular degenerations

Kampik, D ; Basche, M ; Luhmann, U F O ; Nishiguchi, K M ; Williams, J A E ; Greenwood, J ; Moss, S E ; Han, H ; Azam, S ; Duran, Y ; Robbie, S J ; Bainbridge, J W B ; Larkin, D F ; Smith, A J ; Ali, R R

Gene therapy, 2017-12, Vol.24 (12), p.810-818 [Periódico revisado por pares]

England: Nature Publishing Group

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8
Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cells
Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cells
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Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cells

Hoffmann, D ; Schott, J W ; Geis, F K ; Lange, L ; Müller, F-J ; Lenz, D ; Zychlinski, D ; Steinemann, D ; Morgan, M ; Moritz, T ; Schambach, A

Gene therapy, 2017-05, Vol.24 (5), p.298-307 [Periódico revisado por pares]

England: Nature Publishing Group

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9
Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems
Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems
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Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems

Guan, S ; Rosenecker, J

Gene therapy, 2017-03, Vol.24 (3), p.133-143 [Periódico revisado por pares]

England: Nature Publishing Group

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10
The long terminal repeat negative control region is a critical element for insertional oncogenesis after gene transfer into hematopoietic progenitors with Moloney murine leukemia viral vectors
The long terminal repeat negative control region is a critical element for insertional oncogenesis after gene transfer into hematopoietic progenitors with Moloney murine leukemia viral vectors
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The long terminal repeat negative control region is a critical element for insertional oncogenesis after gene transfer into hematopoietic progenitors with Moloney murine leukemia viral vectors

Ikawa, Y ; Uchiyama, T ; Jagadeesh, G J ; Candotti, F

Gene therapy, 2016-11, Vol.23 (11), p.815-818 [Periódico revisado por pares]

England: Nature Publishing Group

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