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1
Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions
Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions
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Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions

Gene therapy, 2021-12, Vol.28 (12), p.683-696 [Peer Reviewed Journal]

2021. The Author(s), under exclusive licence to Springer Nature Limited part of Springer Nature. ;COPYRIGHT 2021 Nature Publishing Group ;The Author(s), under exclusive licence to Springer Nature Limited part of Springer Nature 2021. corrected publication 2021. ;ISSN: 0969-7128 ;EISSN: 1476-5462 ;DOI: 10.1038/s41434-021-00243-z ;PMID: 33658649

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2
How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy
How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy
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How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy

Gene therapy, 2017-09, Vol.24 (9), p.520-526 [Peer Reviewed Journal]

COPYRIGHT 2017 Nature Publishing Group ;COPYRIGHT 2017 Nature Publishing Group ;Copyright Nature Publishing Group Sep 2017 ;Macmillan Publishers Limited, part of Springer Nature. 2017. ;ISSN: 0969-7128 ;EISSN: 1476-5462 ;DOI: 10.1038/gt.2017.34 ;PMID: 28485722

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3
Long noncoding RNA Meg3 regulates cardiomyocyte apoptosis in myocardial infarction
Long noncoding RNA Meg3 regulates cardiomyocyte apoptosis in myocardial infarction
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Long noncoding RNA Meg3 regulates cardiomyocyte apoptosis in myocardial infarction

Gene therapy, 2018-12, Vol.25 (8), p.511-523 [Peer Reviewed Journal]

COPYRIGHT 2018 Nature Publishing Group ;COPYRIGHT 2018 Nature Publishing Group ;Copyright Nature Publishing Group Dec 2018 ;ISSN: 0969-7128 ;EISSN: 1476-5462 ;DOI: 10.1038/s41434-018-0045-4 ;PMID: 30287867

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4
A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases
A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases
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A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases

Gene therapy, 2020-05, Vol.27 (5), p.226-236 [Peer Reviewed Journal]

COPYRIGHT 2020 Nature Publishing Group ;COPYRIGHT 2020 Nature Publishing Group ;The Author(s), under exclusive licence to Springer Nature Limited 2020. ;ISSN: 0969-7128 ;EISSN: 1476-5462 ;DOI: 10.1038/s41434-019-0120-5 ;PMID: 31896760

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5
Toward a new framework for the development of individualized therapies
Toward a new framework for the development of individualized therapies
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Toward a new framework for the development of individualized therapies

Gene therapy, 2021-11, Vol.28 (10-11), p.615-617 [Peer Reviewed Journal]

COPYRIGHT 2021 Nature Publishing Group ;This is a U.S. government work and not under copyright protection in the U.S.; foreign copyright protection may apply. This article is published with open access 2020. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License. ;This is a U.S. government work and not under copyright protection in the U.S.; foreign copyright protection may apply. This article is published with open access 2020 ;ISSN: 0969-7128 ;EISSN: 1476-5462 ;DOI: 10.1038/s41434-020-0143-y ;PMID: 32242078

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6
Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice
Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice
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Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice

Gene therapy, 2021-11, Vol.28 (10-11), p.646-658 [Peer Reviewed Journal]

2021. The Author(s), under exclusive licence to Springer Nature Limited part of Springer Nature. ;COPYRIGHT 2021 Nature Publishing Group ;The Author(s), under exclusive licence to Springer Nature Limited part of Springer Nature 2021. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License. ;The Author(s), under exclusive licence to Springer Nature Limited part of Springer Nature 2021 ;ISSN: 0969-7128 ;EISSN: 1476-5462 ;DOI: 10.1038/s41434-021-00224-2 ;PMID: 33558692

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7
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

Gene therapy, 2021-09, Vol.28 (9), p.494-512 [Peer Reviewed Journal]

2021. The Author(s). ;COPYRIGHT 2021 Nature Publishing Group ;The Author(s) 2021. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License. ;The Author(s) 2021 ;ISSN: 0969-7128 ;EISSN: 1476-5462 ;DOI: 10.1038/s41434-021-00237-x ;PMID: 33753908

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8
The corticotropin releasing hormone-1 (CRH1) receptor antagonist pexacerfont in alcohol dependence: a randomized controlled experimental medicine study
The corticotropin releasing hormone-1 (CRH1) receptor antagonist pexacerfont in alcohol dependence: a randomized controlled experimental medicine study
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The corticotropin releasing hormone-1 (CRH1) receptor antagonist pexacerfont in alcohol dependence: a randomized controlled experimental medicine study

Neuropsychopharmacology (New York, N.Y.), 2015-03, Vol.40 (5), p.1053-1063 [Peer Reviewed Journal]

Copyright Nature Publishing Group Mar 2015 ;Copyright © 2015 American College of Neuropsychopharmacology 2015 American College of Neuropsychopharmacology ;ISSN: 0893-133X ;ISSN: 1740-634X ;EISSN: 1740-634X ;DOI: 10.1038/npp.2014.306 ;PMID: 25409596 ;CODEN: NEROEW

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9
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

Gene therapy, 2020-08, Vol.27 (7-8), p.309-311 [Peer Reviewed Journal]

COPYRIGHT 2020 Nature Publishing Group ;Springer Nature Limited 2020. ;ISSN: 0969-7128 ;EISSN: 1476-5462 ;DOI: 10.1038/s41434-020-0149-5 ;PMID: 32341481

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10
Potent CAR-T cells engineered with Sleeping Beauty transposon vectors display a central memory phenotype
Potent CAR-T cells engineered with Sleeping Beauty transposon vectors display a central memory phenotype
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Potent CAR-T cells engineered with Sleeping Beauty transposon vectors display a central memory phenotype

Gene therapy, 2021-02, Vol.28 (1-2), p.3-5 [Peer Reviewed Journal]

COPYRIGHT 2021 Nature Publishing Group ;Springer Nature Limited 2020. ;ISSN: 0969-7128 ;EISSN: 1476-5462 ;DOI: 10.1038/s41434-020-0138-8 ;PMID: 32139891

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11
Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach
Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach
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Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach

Gene therapy, 2014-01, Vol.21 (1), p.96-105 [Peer Reviewed Journal]

COPYRIGHT 2014 Nature Publishing Group ;COPYRIGHT 2014 Nature Publishing Group ;Copyright Nature Publishing Group Jan 2014 ;ISSN: 0969-7128 ;EISSN: 1476-5462 ;DOI: 10.1038/gt.2013.64 ;PMID: 24225638

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12
ROS in cancer therapy: the bright side of the moon
ROS in cancer therapy: the bright side of the moon
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ROS in cancer therapy: the bright side of the moon

Experimental and Molecular Medicine, 2020, 52(0), , pp.1-12 [Peer Reviewed Journal]

This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License. ;The Author(s) 2020. This work is published under https://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License. ;The Author(s) 2020 ;ISSN: 1226-3613 ;EISSN: 2092-6413 ;DOI: 10.1038/s12276-020-0384-2 ;PMID: 32060354

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13
Identification of leukemia stem cell expression signatures through Monte Carlo feature selection strategy and support vector machine
Identification of leukemia stem cell expression signatures through Monte Carlo feature selection strategy and support vector machine
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Identification of leukemia stem cell expression signatures through Monte Carlo feature selection strategy and support vector machine

Cancer gene therapy, 2020-02, Vol.27 (1-2), p.56-69 [Peer Reviewed Journal]

COPYRIGHT 2020 Nature Publishing Group ;COPYRIGHT 2020 Nature Publishing Group ;Springer Nature America, Inc. 2019. ;ISSN: 0929-1903 ;EISSN: 1476-5500 ;DOI: 10.1038/s41417-019-0105-y ;PMID: 31138902

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14
Gene therapy for cardiovascular diseases in China: basic research
Gene therapy for cardiovascular diseases in China: basic research
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Gene therapy for cardiovascular diseases in China: basic research

Gene therapy, 2020-08, Vol.27 (7-8), p.360-369 [Peer Reviewed Journal]

COPYRIGHT 2020 Nature Publishing Group ;The Author(s), under exclusive licence to Springer Nature Limited 2020. ;ISSN: 0969-7128 ;EISSN: 1476-5462 ;DOI: 10.1038/s41434-020-0148-6 ;PMID: 32341485

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15
Mesenchymal stem cells: environmentally responsive therapeutics for regenerative medicine
Mesenchymal stem cells: environmentally responsive therapeutics for regenerative medicine
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Mesenchymal stem cells: environmentally responsive therapeutics for regenerative medicine

Experimental & molecular medicine, 2013-11, Vol.45 (11), p.e54-e54 [Peer Reviewed Journal]

Copyright Nature Publishing Group Nov 2013 ;Copyright © 2013 KSBMB. 2013 KSBMB. ;ISSN: 2092-6413 ;ISSN: 1226-3613 ;EISSN: 2092-6413 ;DOI: 10.1038/emm.2013.94 ;PMID: 24232253

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16
AAV9-based gene therapy partially ameliorates the clinical phenotype of a mouse model of Leigh syndrome
AAV9-based gene therapy partially ameliorates the clinical phenotype of a mouse model of Leigh syndrome
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AAV9-based gene therapy partially ameliorates the clinical phenotype of a mouse model of Leigh syndrome

Gene therapy, 2017-10, Vol.24 (10), p.661-667 [Peer Reviewed Journal]

COPYRIGHT 2017 Nature Publishing Group ;COPYRIGHT 2017 Nature Publishing Group ;Copyright Nature Publishing Group Oct 2017 ;Copyright © 2017 The Author(s) 2017 The Author(s) ;ISSN: 0969-7128 ;EISSN: 1476-5462 ;DOI: 10.1038/gt.2017.53 ;PMID: 28753212

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17
Artificial intelligence and computational pathology
Artificial intelligence and computational pathology
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Artificial intelligence and computational pathology

Laboratory investigation, 2021-04, Vol.101 (4), p.412-422 [Peer Reviewed Journal]

2021 United States & Canadian Academy of Pathology ;The Author(s), under exclusive licence to United States and Canadian Academy of Pathology 2021. ;The Author(s), under exclusive licence to United States and Canadian Academy of Pathology 2021 ;ISSN: 0023-6837 ;EISSN: 1530-0307 ;DOI: 10.1038/s41374-020-00514-0 ;PMID: 33454724

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18
Extracellular vesicle-based therapeutics: natural versus engineered targeting and trafficking
Extracellular vesicle-based therapeutics: natural versus engineered targeting and trafficking
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Extracellular vesicle-based therapeutics: natural versus engineered targeting and trafficking

Experimental and Molecular Medicine, 2019, 51(0), , pp.1-12 [Peer Reviewed Journal]

This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License. ;The Author(s) 2019 ;ISSN: 2092-6413 ;ISSN: 1226-3613 ;EISSN: 2092-6413 ;DOI: 10.1038/s12276-019-0223-5 ;PMID: 30872574

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19
Proteosomal degradation impairs transcytosis of AAV vectors from suprachoroidal space to retina
Proteosomal degradation impairs transcytosis of AAV vectors from suprachoroidal space to retina
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Proteosomal degradation impairs transcytosis of AAV vectors from suprachoroidal space to retina

Gene therapy, 2021-12, Vol.28 (12), p.740-747 [Peer Reviewed Journal]

2021. The Author(s), under exclusive licence to Springer Nature Limited part of Springer Nature. ;COPYRIGHT 2021 Nature Publishing Group ;The Author(s), under exclusive licence to Springer Nature Limited part of Springer Nature 2021. ;ISSN: 0969-7128 ;EISSN: 1476-5462 ;DOI: 10.1038/s41434-021-00233-1 ;PMID: 33542456

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20
The effect of intranasal insulin on appetite and mood in women with and without obesity: an experimental medicine study
The effect of intranasal insulin on appetite and mood in women with and without obesity: an experimental medicine study
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The effect of intranasal insulin on appetite and mood in women with and without obesity: an experimental medicine study

International Journal of Obesity, 2022-07, Vol.46 (7), p.1319-1327 [Peer Reviewed Journal]

2022. The Author(s). ;The Author(s) 2022. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License. ;The Author(s) 2022 ;ISSN: 0307-0565 ;EISSN: 1476-5497 ;DOI: 10.1038/s41366-022-01115-1 ;PMID: 35397638

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