Approval and therapeutic value of gene therapies in the US and Europe
Gene Therapy, Published online: 08 November 2023; doi:10.1038/s41434-023-00402-4Approval and therapeutic value of gene therapies in the US and Europe (Source: Gene Therapy)
Source: Gene Therapy - November 8, 2023 Category: Genetics & Stem Cells Authors: Kerstin N. Vokinger Camille E. G. Glaus Aaron S. Kesselheim Source Type: research
A practical approach for adoption of a hub and spoke model for cell and gene therapies in low- and middle-income countries: framework and case studies
Gene Therapy, Published online: 30 October 2023; doi:10.1038/s41434-023-00425-xA practical approach for adoption of a hub and spoke model for cell and gene therapies in low- and middle-income countries: framework and case studies (Source: Gene Therapy)
Source: Gene Therapy - October 30, 2023 Category: Genetics & Stem Cells Authors: Shadi Saleh Omar Dabbous Sean D. Sullivan Dipen Ankleshwaria Daiane Trombini Mondher Toumi Mahmoud Diaa Anish Patel Burcu Kazazoglu Taylor Sean Tunis Source Type: research
Implications of maternal-fetal health on perinatal stem cell banking
Gene Therapy, Published online: 26 October 2023; doi:10.1038/s41434-023-00426-wImplications of maternal-fetal health on perinatal stem cell banking (Source: Gene Therapy)
Source: Gene Therapy - October 26, 2023 Category: Genetics & Stem Cells Authors: Dandan Zhu Mehri Barabadi Courtney McDonald Gina Kusuma Ishmael Miguel Inocencio Rebecca Lim Source Type: research
p53 dry gene powder enhances anti-cancer effects of chemotherapy against malignant pleural mesothelioma
Gene Therapy, Published online: 13 October 2023; doi:10.1038/s41434-023-00424-yp53 dry gene powder enhances anti-cancer effects of chemotherapy against malignant pleural mesothelioma (Source: Gene Therapy)
Source: Gene Therapy - October 13, 2023 Category: Genetics & Stem Cells Authors: Naomi Muramatsu Misa Ichikawa Tomoko Katagiri Yumi Taguchi Takashi Hatanaka Tomoyuki Okuda Hirokazu Okamoto Source Type: research
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques
Gene Therapy, Published online: 13 October 2023; doi:10.1038/s41434-023-00423-zPrednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques (Source: Gene Therapy)
Source: Gene Therapy - October 13, 2023 Category: Genetics & Stem Cells Authors: Alexander Kistner Jessica A. Chichester Lili Wang Roberto Calcedo Jenny A. Greig Leah N. Cardwell Margaret C. Wright Julien Couthouis Sunjay Sethi Brian E. McIntosh Kathleen McKeever Samuel Wadsworth James M. Wilson Emil Kakkis Barbara A. Sullivan Source Type: research
Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo
Gene Therapy, Published online: 26 September 2023; doi:10.1038/s41434-023-00418-wArtificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo (Source: Gene Therapy)
Source: Gene Therapy - September 26, 2023 Category: Genetics & Stem Cells Authors: Gabriela Toro Cabrera Katharina E. Meijboom Abbas Abdallah Helene Tran Zachariah Foster Alexandra Weiss Nicholas Wightman Rachel Stock Tania Gendron Alisha Gruntman Anthony Giampetruzzi Leonard Petrucelli Robert H. Brown Jr Christian Mueller Source Type: research
Long-term effects of a fat-directed FGF21 gene therapy in aged female mice
Gene Therapy, Published online: 12 September 2023; doi:10.1038/s41434-023-00422-0Long-term effects of a fat-directed FGF21 gene therapy in aged female mice (Source: Gene Therapy)
Source: Gene Therapy - September 12, 2023 Category: Genetics & Stem Cells Authors: Jacqueline M. Anderson W. David Arnold Wei Huang Alissa Ray Gregory Owendoff Lei Cao Source Type: research
Acoustically targeted noninvasive gene therapy in large brain volumes
Gene Therapy, Published online: 12 September 2023; doi:10.1038/s41434-023-00421-1Acoustically targeted noninvasive gene therapy in large brain volumes (Source: Gene Therapy)
Source: Gene Therapy - September 12, 2023 Category: Genetics & Stem Cells Authors: Shirin Nouraein Sangsin Lee Vidal A. Saenz Huckie C. Del Mundo Joycelyn Yiu Jerzy O. Szablowski Source Type: research
A pilot study to determine the optimal dose of scAAVIL-1ra in a large animal model of post-traumatic osteoarthritis
Gene Therapy, Published online: 11 September 2023; doi:10.1038/s41434-023-00420-2A pilot study to determine the optimal dose of scAAVIL-1ra in a large animal model of post-traumatic osteoarthritis (Source: Gene Therapy)
Source: Gene Therapy - September 11, 2023 Category: Genetics & Stem Cells Authors: P. Thampi K. A. Seabaugh L. M. Pezzanite C. R. Chu J. N. Phillips J. C. Grieger C. W. McIlwraith R. J. Samulski L. R. Goodrich Source Type: research
Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic
Gene Therapy, Published online: 23 August 2023; doi:10.1038/s41434-023-00417-xSelection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic (Source: Gene Therapy)
Source: Gene Therapy - August 23, 2023 Category: Genetics & Stem Cells Authors: Mark Singh Andrew Brooks Parto Toofan Keith McLuckie Source Type: research
Safety and biodistribution of XC001 (encoberminogene rezmadenovec) gene therapy in rats: a potential therapy for cardiovascular diseases
Gene Therapy, Published online: 18 August 2023; doi:10.1038/s41434-023-00416-ySafety and biodistribution of XC001 (encoberminogene rezmadenovec) gene therapy in rats: a potential therapy for cardiovascular diseases (Source: Gene Therapy)
Source: Gene Therapy - August 18, 2023 Category: Genetics & Stem Cells Authors: Duncan J. Stewart Albert Gianchetti Dawn Byrnes Howard C. Dittrich Barb Thorne Linda L. Manza Rickey R. Reinhardt Source Type: research
Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates
Gene Therapy, Published online: 17 August 2023; doi:10.1038/s41434-023-00410-4Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates (Source: Gene Therapy)
Source: Gene Therapy - August 17, 2023 Category: Genetics & Stem Cells Authors: Tricia H. Burdo Chen Chen Rafal Kaminski Ilker K. Sariyer Pietro Mancuso Martina Donadoni Mandy D. Smith Rahsan Sariyer Maurizio Caocci Shuren Liao Hong Liu Wenwen Huo Huaqing Zhao John Misamore Mark G. Lewis Vahan Simonyan Ethan Y. Xu Thomas J. Cradick J Source Type: research
Biodistribution of lentiviral transduced adipose-derived stem cells for “ex-vivo” regional gene therapy for bone repair
Gene Therapy, Published online: 11 August 2023; doi:10.1038/s41434-023-00415-zBiodistribution of lentiviral transduced adipose-derived stem cells for “ex-vivo” regional gene therapy for bone repair (Source: Gene Therapy)
Source: Gene Therapy - August 11, 2023 Category: Genetics & Stem Cells Authors: Jennifer A. Bell Kevin Collon Cory Mayfield Matthew C. Gallo Stephanie W. Chang Osamu Sugiyama Amy H. Tang Roger Hollis Shefali Chopra Donald B. Kohn Jay R. Lieberman Source Type: research
Targeted shock-and-kill HIV-1 gene therapy approach combining CRISPR activation, suicide gene tBid and retargeted adenovirus delivery
Gene Therapy, Published online: 09 August 2023; doi:10.1038/s41434-023-00413-1Targeted shock-and-kill HIV-1 gene therapy approach combining CRISPR activation, suicide gene tBid and retargeted adenovirus delivery (Source: Gene Therapy)
Source: Gene Therapy - August 9, 2023 Category: Genetics & Stem Cells Authors: Sarah Klinnert Corinne D. Schenkel Patrick C. Freitag Huldrych F. G ünthard Andreas Pl ückthun Karin J. Metzner Source Type: research
CRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´s disease
Gene Therapy, Published online: 04 August 2023; doi:10.1038/s41434-023-00414-0CRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´s disease (Source: Gene Therapy)
Source: Gene Therapy - August 4, 2023 Category: Genetics & Stem Cells Authors: Luis Fernando Narv áez-Pérez Francisco Paz-Berm údez Jos é Arturo Avalos-Fuentes Aurelio Campos-Romo Benjam ín Florán-Garduño Jos é Segovia Source Type: research
