Conditional Immortalization of Lymphoid Progenitors via Tetracycline-Regulated LMO2 Expression
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 24, 2020 Category: Genetics & Stem Cells Authors: Ekaterina Koniaeva Maike Stahlhut Lucas Lange Martin G. Sauer Olga S. Kustikova Axel Schambach Source Type: research

Transduction Efficiency of Adeno-Associated Virus Serotypes After Local Injection in Mouse and Human Skeletal Muscle
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 24, 2020 Category: Genetics & Stem Cells Authors: Laura Muraine Mona Bensalah Jamila Dhiab Gonzalo Cordova Ludovic Arandel Alix Marhic Maud Chapart St éphane Vasseur Sofia Benkhelifa-Ziyyat Anne Bigot Gillian Butler-Browne Vincent Mouly Elisa Negroni Capucine Trollet Source Type: research

Aberrantly Expressed Timeless Regulates Cell Proliferation and Cisplatin Efficacy in Cervical Cancer
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 24, 2020 Category: Genetics & Stem Cells Authors: Jinhua Zhou Yinghui Zhang Xinwei Zou Lingling Kuai Li Wang Juan Wang Fangrong Shen Jinghui Hu Xia Zhang Yazhen Huang Youguo Chen Source Type: research

Differential Transgene Silencing of Myeloid-Specific Promoters in the AAVS1 Safe Harbor Locus of Induced Pluripotent Stem Cell-Derived Myeloid Cells
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 23, 2020 Category: Genetics & Stem Cells Authors: Denise Klatt Erica Cheng Dirk Hoffmann Giorgia Santilli Adrian J. Thrasher Christian Brendel Axel Schambach Source Type: research

Get Smart About Home-Cage Monitoring
Human Gene Therapy,Volume 31, Issue 1-2, Page 129-129, January 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 21, 2020 Category: Genetics & Stem Cells Source Type: research

Period 2-Induced Activation of Autophagy Improves Cardiac Remodeling After Myocardial Infarction
Human Gene Therapy,Volume 31, Issue 1-2, Page 119-128, January 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 21, 2020 Category: Genetics & Stem Cells Authors: Xin-Yu Zhang Lin Wang Wen-Jiang Yan Xiao-Ting Lu Xin-Yun Li Yuan-Yuan Sun Source Type: research

Specific Overexpression of Mitofusin-2 in Hepatic Stellate Cells Ameliorates Liver Fibrosis in Mice Model
Human Gene Therapy,Volume 31, Issue 1-2, Page 103-109, January 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 21, 2020 Category: Genetics & Stem Cells Authors: Hanzhang Zhu Yuqiang Shan Ke Ge Jun Lu Wencheng Kong Changku Jia Source Type: research

Public Acceptability of Gene Therapy and Gene Editing for Human Use: A Systematic Review
Human Gene Therapy,Volume 31, Issue 1-2, Page 20-46, January 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 21, 2020 Category: Genetics & Stem Cells Authors: Juliette Delhove Ivana Osenk Ivanka Prichard Martin Donnelley Source Type: research

Translational Medicine: Evolution, Fulfillment, and Belief in Gene Therapy
Human Gene Therapy,Volume 31, Issue 1-2, Page 14-19, January 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 21, 2020 Category: Genetics & Stem Cells Authors: Jerry R. Mendell Source Type: research

Gene Therapy Untangles the Problem of Chronic Traumatic Encephalopathy
Human Gene Therapy,Volume 31, Issue 1-2, Page 12-13, January 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 21, 2020 Category: Genetics & Stem Cells Authors: Terence R. Flotte Source Type: research

Gene Therapy on the Ascent
Human Gene Therapy,Volume 31, Issue 1-2, Page 9-11, January 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 21, 2020 Category: Genetics & Stem Cells Source Type: research

30 Years Later —Reflections on Human Gene Therapy: An Interview with Terry Flotte, MD
Human Gene Therapy,Volume 31, Issue 1-2, Page 5-8, January 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 21, 2020 Category: Genetics & Stem Cells Authors: Kevin Davies Source Type: research

Ringing in the Changes
Human Gene Therapy,Volume 31, Issue 1-2, Page 4-4, January 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 21, 2020 Category: Genetics & Stem Cells Authors: Terence R. Flotte Guangping Gao Source Type: research

2020: Gene Therapy Enters Its Fourth Decade
Human Gene Therapy,Volume 31, Issue 1-2, Page 2-3, January 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 21, 2020 Category: Genetics & Stem Cells Authors: Terence R. Flotte Guangping Gao Source Type: research

Applications of Next Generation CRISPR-derived Technologies
Human Gene Therapy,Volume 31, Issue 1-2, Page 1-1, January 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 21, 2020 Category: Genetics & Stem Cells Source Type: research

Exosome-Mediated Transfer of Anti-miR-33a-5p from Transduced Endothelial Cells Enhances Macrophage and Vascular Smooth Muscle Cell Cholesterol Efflux
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 16, 2020 Category: Genetics & Stem Cells Authors: Alexis Stamatikos Ethan Knight Lucia Vojtech Lianxiang Bi Bradley K. Wacker Chongren Tang David A. Dichek Source Type: research

Sexually Divergent Mortality and Partial Phenotypic Rescue After Gene Therapy in a Mouse Model of Dravet Syndrome
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 16, 2020 Category: Genetics & Stem Cells Authors: Yosuke Niibori Shiron J. Lee Berge A. Minassian David R. Hampson Source Type: research

Restoration of Functional Full-Length Dystrophin After Intramuscular Transplantation of Foamy Virus-Transduced Myoblasts
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 10, 2020 Category: Genetics & Stem Cells Authors: Jinhong Meng Nathan Paul Sweeney Bruno Doreste Francesco Muntoni Myra McClure Jennifer Morgan Source Type: research

Can Adeno-Associated Viral Vectors Deliver Effectively Large Genes?
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 9, 2020 Category: Genetics & Stem Cells Authors: Patrizia Tornabene Ivana Trapani Source Type: research

Systemic Safety of a Recombinant AAV8 Vector for Human Cocaine Hydrolase Gene Therapy: A Good Laboratory Practice Preclinical Study in Mice
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - December 19, 2019 Category: Genetics & Stem Cells Authors: Vicky Ping Chen Yang Gao Liyi Geng Mike Steele Nathan Jenks Kah-Whye Peng Stephen Brimijoin Source Type: research

Sustained Interleukin-10 Transgene Expression Following Intra-Articular AAV5-IL-10 Administration to Horses
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - December 18, 2019 Category: Genetics & Stem Cells Authors: Kaitlyn L. Moss Zibin Jiang Michael E. Dodson Renata L. Linardi Joanne Haughan Alexis L. Gale Cara Grzybowski Julie E. Engiles Darko Stefanovski Mary A. Robinson Kyla F. Ortved Source Type: research

Long-Term Correction of Copper Metabolism in Wilson's Disease Mice with AAV8 Vector Delivering Truncated ATP7B
Human Gene Therapy,Volume 30, Issue 12, Page 1494-1504, December 2019. (Source: Human Gene Therapy)
Source: Human Gene Therapy - December 16, 2019 Category: Genetics & Stem Cells Authors: Yingying Leng Ping Li Lifang Zhou Lin Xiao Yu Liu Zhaoyue Zheng Fengming Qin Qiukui Hao Heng Xu Shaohua Yao Biao Dong Source Type: research

Recombinant Adeno-Associated Virus Quality Control for Non-Clinical and Clinical Vectors: How an Unregulated Commercial Sector Can Compromise Development of New Gene Therapies
Human Gene Therapy,Volume 30, Issue 12, Page 1447-1448, December 2019. (Source: Human Gene Therapy)
Source: Human Gene Therapy - December 16, 2019 Category: Genetics & Stem Cells Authors: Robert M. Kotin J. Fraser Wright Source Type: research

Prime Editing: A Novel Cas9-Reverse Transcriptase Fusion May Revolutionize Genome Editing
Human Gene Therapy,Volume 30, Issue 12, Page 1445-1446, December 2019. (Source: Human Gene Therapy)
Source: Human Gene Therapy - December 16, 2019 Category: Genetics & Stem Cells Authors: Terence R. Flotte Guangping Gao Source Type: research

Ly6a Differential Expression in Blood –Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - December 14, 2019 Category: Genetics & Stem Cells Authors: Ana Rita Batista Oliver D. King Christopher P. Reardon Crystal Davis Shankaracharya Vivek Philip Heather Gray-Edwards Neil Aronin Cathleen Lutz John Landers Miguel Sena-Esteves Source Type: research

Anti-Phospho-Tau Gene Therapy for Chronic Traumatic Encephalopathy
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - December 14, 2019 Category: Genetics & Stem Cells Authors: Chester Bittencourt Sacramento Dolan Sondhi Jonathan B. Rosenberg Alvin Chen Stephanie Giordano Eduard Pey Vladlena Lee Katie M. Stiles David F. Havlicek Philip L. Leopold Stephen M. Kaminsky Ronald G. Crystal Source Type: research

A Common Outer Retinal Change in Retinal Degeneration by Optical Coherence Tomography Can Be Used to Assess Outcomes of Gene Therapy
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - December 5, 2019 Category: Genetics & Stem Cells Authors: Myung Kuk Joe Wenbo Li Suja Hiriyanna Wenhan Yu Shreya A. Shah Mones Abu-Asab Haohua Qian Zhijian Wu Source Type: research

Vectofusin-1 Improves Transduction of Primary Human Cells with Diverse Retroviral and Lentiviral Pseudotypes, Enabling Robust, Automated Closed-System Manufacturing
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - December 5, 2019 Category: Genetics & Stem Cells Authors: Constanze Radek Ornellie Bernadin Katharina Drechsel Nicole Cordes Rita Pfeifer Pia Str äßer Mirella Mormin Alejandra Gutierrez-Guerrero Fran çois-loïc Cosset Andrew D. Kaiser Thomas Schaser Anne Galy Els Verhoeyen Ian C.D. Johnston Source Type: research

Comparison of Gene Delivery to the Kidney by Adenovirus, Adeno-Associated Virus, and Lentiviral Vectors After Intravenous and Direct Kidney Injections
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - December 5, 2019 Category: Genetics & Stem Cells Authors: Jeffrey D. Rubin Tien V. Nguyen Kari L. Allen Katayoun Ayasoufi Michael A. Barry Source Type: research

Rational Engineering and Preclinical Evaluation of Neddylation and SUMOylation Site Modified Adeno-Associated Virus Vectors in Murine Models of Hemophilia B and Leber Congenital Amaurosis
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 27, 2019 Category: Genetics & Stem Cells Authors: Shubham Maurya Bertin Mary Giridhara R. Jayandharan Source Type: research

Correction to: Single Plasmid-Based, Upgradable, and Backward-Compatible Adenoviral Vector Systems, by Liu H, Lu Z, Zhang X, Guo X, Mei L, Zou X, Zhong Y, Wang M, and Hung T. Hum Gene Ther 2019:30(6):777 –791.DOI: 10.1089/hum.2018.258
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 19, 2019 Category: Genetics & Stem Cells Source Type: research

Filling Adeno-Associated Virus Capsids: Estimating Success by Cryo-Electron Microscopy
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 14, 2019 Category: Genetics & Stem Cells Authors: Suriyasri Subramanian Anna C. Maurer Carol M. Bator Alexander M. Makhov James F. Conway Kevin B. Turner James H. Marden Luk H. Vandenberghe Susan L. Hafenstein Source Type: research

Silencing Breast Cancer Genes Using Morpholino Embedded DNA-Tile-AuNPs Nanostructures
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 13, 2019 Category: Genetics & Stem Cells Authors: Cansu U. Tun ç Deniz Y. Öztaş Deniz Uzuno ğlu Ömer F. Bayrak Mustafa Çulha Source Type: research

Silencing of GAS5 Alleviates Glaucoma in Rat Models by Reducing Retinal Ganglion Cell Apoptosis
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 13, 2019 Category: Genetics & Stem Cells Authors: Rong-Rong Zhou Hai-Bo Li Qi-Sheng You Rong Rong Meng-Ling You Kun Xiong Ju-Fang Huang Xiao-Bo Xia Dan Ji Source Type: research

Correction to: Effect of Human Wnt10b Transgene Overexpression on Peri-Implant Osteogenesis in Ovariectomized Rats by Liu H, Zhang N, Liu Y, Liu L, Yin G, and En L. Hum Gene Ther 2018;29:1416 –1427. DOI: 10.1089/hum.2018.003
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - November 5, 2019 Category: Genetics & Stem Cells Source Type: research

Ocular Inflammatory Response to Intravitreal Injection of Adeno-Associated Virus Vector: Relative Contribution of Genome and Capsid
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - October 31, 2019 Category: Genetics & Stem Cells Authors: Adrian M. Timmers Judith A. Newmark Heikki T. Turunen Tanaz Farivar Jilin Liu Chunjuan Song Guo-jie Ye Steven Pennock Chantelle Gaskin David R. Knop Mark S. Shearman Source Type: research

Breathing New Life into TRAIL for Breast Cancer Therapy: Co-Delivery of pTRAIL and Complementary siRNAs Using Lipopolymers
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - October 29, 2019 Category: Genetics & Stem Cells Authors: Bindu Thapa Remant KC Markian Bahniuk Janine Schmitke Mary Hitt Afsaneh Lavasanifar Olaf Kutsch Dai-Wu Seol Hasan Uludag Source Type: research

Utility of Self-Destructing CRISPR/Cas Constructs for Targeted Gene Editing in the Retina
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - October 25, 2019 Category: Genetics & Stem Cells Authors: Fan Li Sandy S.C. Hung Mohd Khairul Nizam Mohd Khalid Jiang-Hui Wang Vicki Chrysostomou Vickie H.Y. Wong Vikrant Singh Kristof Wing Leilei Tu James A. Bender Alice P ébay Anna E. King Anthony L. Cook Raymond C.B. Wong Bang V. Bui Alex W. Hewitt Guei-Sheu Source Type: research

ESGCT 27th Annual Congress In collaboration with SETGyc Abstract Author Index
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - October 21, 2019 Category: Genetics & Stem Cells Source Type: research

ESGCT 27th Annual Congress In collaboration with SETGyc Barcelona, Spain October 22 –25, 2019 Abstracts
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - October 21, 2019 Category: Genetics & Stem Cells Source Type: research

Dual ABCA4-AAV Vector Treatment Reduces Pathogenic Retinal A2E Accumulation in a Mouse Model of Autosomal Recessive Stargardt Disease
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 30, 2019 Category: Genetics & Stem Cells Authors: Frank M. Dyka Laurie L. Molday Vince A. Chiodo Robert S. Molday William W. Hauswirth Source Type: research

Helper-Dependent Adenovirus Transduces the Human and Rat Retina but Elicits an Inflammatory Reaction When Delivered Subretinally in Rats
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 26, 2019 Category: Genetics & Stem Cells Authors: Ian C. Han Erin R. Burnight Mallory J. Ulferts Kristan S. Worthington Stephen R. Russell Elliott H. Sohn Robert F. Mullins Edwin M. Stone Budd A. Tucker Luke A. Wiley Source Type: research

Nonviral Delivery of GRIM-19 Gene Inhibits Tumor Growth with Reduced Local and Systemic Complications
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 25, 2019 Category: Genetics & Stem Cells Authors: Yang Xu Xinghuo Wang Baofeng Guo Ding Wang Dhan V. Kalvakolanu Xuyang Chen Jun Tang Ling Zhang Qing Yang Source Type: research

Gene Delivery to Human Limbal Stem Cells Using Viral Vectors
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 25, 2019 Category: Genetics & Stem Cells Authors: Liujiang Song Zhenwei Song Nathaniel J. Fry Laura Conatser Telmo Llanga Hua Mei Tal Kafri Matthew L. Hirsch Source Type: research

Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler –Najjar Syndrome
Human Gene Therapy,Volume 30, Issue 10, Page 1297-1305, October 2019. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 24, 2019 Category: Genetics & Stem Cells Authors: Sem J. Aronson Philippe Veron Fanny Collaud Aur élie Hubert Virginie Delahais G éraldine Honnet Robert J. de Knegt Norman Junge Ulrich Baumann Angelo Di Giorgio Lorenzo D'Antiga Virginia M. Ginocchio Nicola Brunetti-Pierri Philippe Labrune Ulrich Beuers Source Type: research

In Vivo Gene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon
Human Gene Therapy,Volume 30, Issue 10, Page 1211-1221, October 2019. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 24, 2019 Category: Genetics & Stem Cells Authors: Sara Marc ó Virginia Haurigot Fatima Bosch Source Type: research

Genetic-Based Approaches to Inherited Metabolic Liver Diseases
Human Gene Therapy,Volume 30, Issue 10, Page 1190-1203, October 2019. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 24, 2019 Category: Genetics & Stem Cells Authors: Nerea Zabaleta Mirja Hommel David Salas Gloria Gonzalez-Aseguinolaza Source Type: research

Breaking the Barriers of Genetic and Metabolic Disorders
Human Gene Therapy,Volume 30, Issue 10, Page 1177-1179, October 2019. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 24, 2019 Category: Genetics & Stem Cells Authors: Hildegard B üning Fatima Bosch Federico Mingozzi Source Type: research

Capsid Engineering Overcomes Barriers Toward Adeno-Associated Virus Vector-Mediated Transduction of Endothelial Cells
Human Gene Therapy,Volume 30, Issue 10, Page 1284-1296, October 2019. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 23, 2019 Category: Genetics & Stem Cells Authors: L. Zhang A. Rossi L. Lange N. Meumann U. Koitzsch K. Christie M.A. Nesbit C.B.T. Moore U.T. Hacker M. Morgan D. Hoffmann J. Zengel J.E. Carette A. Schambach A. Salvetti M. Odenthal H. B üning Source Type: research

Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 13, 2019 Category: Genetics & Stem Cells Authors: Virginia Maria Ginocchio Rita Ferla Alberto Auricchio Nicola Brunetti-Pierri Source Type: research