Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders
Gene Therapy, Published online: 09 March 2021; doi:10.1038/s41434-021-00247-9Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders (Source: Gene Therapy)
Source: Gene Therapy - March 9, 2021 Category: Genetics & Stem Cells Authors: Yan Chen Ruiting Wen Zhigang Yang Zhanghui Chen Source Type: research
Mesenchymal stem cells overexpressing BMP-9 by CRISPR-Cas9 present high in vitro osteogenic potential and enhance in vivo bone formation
Gene Therapy, Published online: 08 March 2021; doi:10.1038/s41434-021-00248-8Mesenchymal stem cells overexpressing BMP-9 by CRISPR-Cas9 present high in vitro osteogenic potential and enhance in vivo bone formation (Source: Gene Therapy)
Source: Gene Therapy - March 8, 2021 Category: Genetics & Stem Cells Authors: Gileade P. Freitas Helena B. Lopes Alann T. P. Souza Maria Paula O. Gomes Georgia K. Quiles Jonathan Gordon Coralee Tye Janet L. Stein Gary S. Stein Jane B. Lian Marcio M. Beloti Adalberto L. Rosa Source Type: research
Alternative oxidase encoded by sequence-optimized and chemically-modified RNA transfected into mammalian cells is catalytically active
Gene Therapy, Published online: 04 March 2021; doi:10.1038/s41434-021-00235-zAlternative oxidase encoded by sequence-optimized and chemically-modified RNA transfected into mammalian cells is catalytically active (Source: Gene Therapy)
Source: Gene Therapy - March 4, 2021 Category: Genetics & Stem Cells Authors: Luca Giordano Manish K. Aneja Natascha Sommer Nasim Alebrahimdehkordi Alireza Seraji Norbert Weissmann Carsten Rudolph Christian Plank Howard T. Jacobs Marten Szibor Source Type: research
Correction: Implications of hematopoietic stem cells heterogeneity for gene therapies
Gene Therapy, Published online: 04 March 2021; doi:10.1038/s41434-021-00245-xCorrection: Implications of hematopoietic stem cells heterogeneity for gene therapies (Source: Gene Therapy)
Source: Gene Therapy - March 4, 2021 Category: Genetics & Stem Cells Authors: Jeremy Epah Richard Sch äfer Source Type: research
Ectopic expression of BBS1 rescues male infertility, but not retinal degeneration, in a BBS1 mouse model
Gene Therapy, Published online: 04 March 2021; doi:10.1038/s41434-021-00241-1Ectopic expression of BBS1 rescues male infertility, but not retinal degeneration, in a BBS1 mouse model (Source: Gene Therapy)
Source: Gene Therapy - March 4, 2021 Category: Genetics & Stem Cells Authors: Matthew R. Cring Kacie J. Meyer Charles C. Searby Adam Hedberg-Buenz Michael Cave Michael G. Anderson Kai Wang Val C. Sheffield Source Type: research
Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions
Gene Therapy, Published online: 03 March 2021; doi:10.1038/s41434-021-00243-zIntracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions (Source: Gene Therapy)
Source: Gene Therapy - March 3, 2021 Category: Genetics & Stem Cells Authors: Jalish M. Riyad Thomas Weber Source Type: research
Fabrication of anionic dextran-coated micelles for aptamer targeted delivery of camptothecin and survivin-shRNA to colon adenocarcinoma
Gene Therapy, Published online: 25 February 2021; doi:10.1038/s41434-021-00234-0Fabrication of anionic dextran-coated micelles for aptamer targeted delivery of camptothecin and survivin-shRNA to colon adenocarcinoma (Source: Gene Therapy)
Source: Gene Therapy - February 25, 2021 Category: Genetics & Stem Cells Authors: Setareh Sanati Sahar Taghavi Khalil Abnous Seyed Mohammad Taghdisi Maryam Babaei Mohammad Ramezani Mona Alibolandi Source Type: research
Genetic insights, disease mechanisms, and biological therapeutics for Waardenburg syndrome
Gene Therapy, Published online: 25 February 2021; doi:10.1038/s41434-021-00240-2Genetic insights, disease mechanisms, and biological therapeutics for Waardenburg syndrome (Source: Gene Therapy)
Source: Gene Therapy - February 25, 2021 Category: Genetics & Stem Cells Authors: Sida Huang Jian Song Chufeng He Xinzhang Cai Kai Yuan Lingyun Mei Yong Feng Source Type: research
Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review
Gene Therapy, Published online: 22 February 2021; doi:10.1038/s41434-021-00238-wInsights and future directions of potential genetic therapy for Apert syndrome: A systematic review (Source: Gene Therapy)
Source: Gene Therapy - February 22, 2021 Category: Genetics & Stem Cells Authors: Nisreen Mohammed Al-Namnam Soher Nagi Jayash Firdaus Hariri Zainal Ariff Abdul Rahman Mohammed Abdullah Alshawsh Source Type: research
Use of CRISPR/Cas9-mediated disruption of CNS cell type genes to profile transduction of AAV by neonatal intracerebroventricular delivery in mice
Gene Therapy, Published online: 22 February 2021; doi:10.1038/s41434-021-00223-3Use of CRISPR/Cas9-mediated disruption of CNS cell type genes to profile transduction of AAV by neonatal intracerebroventricular delivery in mice (Source: Gene Therapy)
Source: Gene Therapy - February 22, 2021 Category: Genetics & Stem Cells Authors: Tess Torregrosa Sydney Lehman Sam Hana Galina Marsh Shanqin Xu Kathryn Koszka Nicole Mastrangelo Alexander McCampbell Christopher E. Henderson Shih-Ching Lo Source Type: research
AAV-mediated delivery of actoxumab and bezlotoxumab results in serum and mucosal antibody concentrations that provide protection from C. difficile toxin challenge
Gene Therapy, Published online: 19 February 2021; doi:10.1038/s41434-021-00236-yAAV-mediated delivery of actoxumab and bezlotoxumab results in serum and mucosal antibody concentrations that provide protection from C. difficile toxin challenge (Source: Gene Therapy)
Source: Gene Therapy - February 19, 2021 Category: Genetics & Stem Cells Authors: Matthew M. Guilleman Brenna A. Y. Stevens Laura P. Van Lieshout Amira D. Rghei Yanlong Pei Lisa A. Santry Brad Thompson Sarah K. Wootton Source Type: research
Rescue of retinal ganglion cells in optic nerve injury using cell-selective AAV mediated delivery of SIRT1
Gene Therapy, Published online: 15 February 2021; doi:10.1038/s41434-021-00219-zRescue of retinal ganglion cells in optic nerve injury using cell-selective AAV mediated delivery of SIRT1 (Source: Gene Therapy)
Source: Gene Therapy - February 15, 2021 Category: Genetics & Stem Cells Authors: Ahmara G. Ross Devin S. McDougald Reas S. Khan Thu T. Duong Kimberly E. Dine Puya Aravand Jean Bennett Venkata Ramana Murthy Chavali Kenneth S. Shindler Source Type: research
Implications of hematopoietic stem cells heterogeneity for gene therapies
Gene Therapy, Published online: 15 February 2021; doi:10.1038/s41434-021-00229-xImplications of hematopoietic stem cells heterogeneity for gene therapies (Source: Gene Therapy)
Source: Gene Therapy - February 15, 2021 Category: Genetics & Stem Cells Authors: Jeremy Epah Richard Sch äfer Source Type: research
Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?
Gene Therapy, Published online: 11 February 2021; doi:10.1038/s41434-021-00232-2Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy? (Source: Gene Therapy)
Source: Gene Therapy - February 11, 2021 Category: Genetics & Stem Cells Authors: Dimitrios Laurin Wagner Lena Peter Michael Schmueck-Henneresse Source Type: research
Gene delivery to the hypoglossal motor system: preclinical studies and translational potential
Gene Therapy, Published online: 11 February 2021; doi:10.1038/s41434-021-00225-1Gene delivery to the hypoglossal motor system: preclinical studies and translational potential (Source: Gene Therapy)
Source: Gene Therapy - February 11, 2021 Category: Genetics & Stem Cells Authors: Brendan M. Doyle Michele L. Singer Thomaz Fleury-Curado Sabhya Rana Ethan S. Benevides Barry J. Byrne Vsevolod Y. Polotsky David D. Fuller Source Type: research
