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
Sildenafil increases AAV9 transduction after a systemic administration and enhances AAV9-dystrophin therapeutic effect in mdx mice
Gene Therapy, Published online: 27 July 2023; doi:10.1038/s41434-023-00411-3Sildenafil increases AAV9 transduction after a systemic administration and enhances AAV9-dystrophin therapeutic effect in mdx mice (Source: Gene Therapy)
Source: Gene Therapy - July 27, 2023 Category: Genetics & Stem Cells Authors: Kaiyi Zhou Meng Yuan Jiabao Sun Feixu Zhang Xiaoying Zong Zhanao Li Dingyue Tang Lichen Zhou Jing Zheng Xiao Xiao Xia Wu Source Type: research
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse
Gene Therapy, Published online: 27 July 2023; doi:10.1038/s41434-023-00412-2Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse (Source: Gene Therapy)
Source: Gene Therapy - July 27, 2023 Category: Genetics & Stem Cells Authors: Janine A. Gilkes Benjamin L. Judkins Brontie N. Herrera Ronald J. Mandel Sanford L. Boye Shannon E. Boye Arun Srivastava Coy D. Heldermon Source Type: research
Immunobiology of a rationally-designed AAV2 capsid following intravitreal delivery in mice
Gene Therapy, Published online: 29 June 2023; doi:10.1038/s41434-023-00409-xImmunobiology of a rationally-designed AAV2 capsid following intravitreal delivery in mice (Source: Gene Therapy)
Source: Gene Therapy - June 29, 2023 Category: Genetics & Stem Cells Authors: Michael Whitehead Andrew Sage Tom Burgoyne Andrew Osborne Patrick Yu-Wai-Man Keith R. Martin Source Type: research
Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo
Gene Therapy, Published online: 15 June 2023; doi:10.1038/s41434-023-00406-0Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo (Source: Gene Therapy)
Source: Gene Therapy - June 15, 2023 Category: Genetics & Stem Cells Authors: Neda A. M. Nafchi Ellie M. Chilcott Sharon Brown Heidi R. Fuller Melissa Bowerman Rafael J. Y áñez-Muñoz Source Type: research
Tolerability and tropism of recombinant adeno-associated virus vectors in the African green monkey (Chlorocebus sabaeus) anterior chamber
Gene Therapy, Published online: 24 May 2023; doi:10.1038/s41434-023-00407-zTolerability and tropism of recombinant adeno-associated virus vectors in the African green monkey (Chlorocebus sabaeus) anterior chamber (Source: Gene Therapy)
Source: Gene Therapy - May 24, 2023 Category: Genetics & Stem Cells Authors: Kristina J. Chern Kimicia Z. Issac Zendorf D. Gumbs Merissa E. O ’Connor Matthew S. Lawrence Daniel M. Lipinski Source Type: research
RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells
Gene Therapy, Published online: 12 May 2023; doi:10.1038/s41434-023-00404-2RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells (Source: Gene Therapy)
Source: Gene Therapy - May 12, 2023 Category: Genetics & Stem Cells Authors: Lucie Bouquet Elodie B ôle-Richard Walid Warda Mathieu Neto Da Rocha Rim Trad Cl émentine Nicod Rafik Haderbache Delphine Genin Christophe Ferrand Marina Deschamps Source Type: research
Adeno-associated virus vectors and neurotoxicity—lessons from preclinical and human studies
Gene Therapy, Published online: 10 May 2023; doi:10.1038/s41434-023-00405-1Adeno-associated virus vectors and neurotoxicity—lessons from preclinical and human studies (Source: Gene Therapy)
Source: Gene Therapy - May 10, 2023 Category: Genetics & Stem Cells Authors: Daniel Stone Martine Aubert Keith R. Jerome Source Type: research
Repeat or single-dose lentiviral vector administration to mouse lungs? It’s all about the timing
Gene Therapy, Published online: 10 May 2023; doi:10.1038/s41434-023-00403-3Repeat or single-dose lentiviral vector administration to mouse lungs? It’s all about the timing (Source: Gene Therapy)
Source: Gene Therapy - May 10, 2023 Category: Genetics & Stem Cells Authors: Martin Donnelley Patricia Cmielewski Emma Knight Chantelle Carpentieri Alexandra McCarron Nathan Rout-Pitt David Parsons Nigel Farrow Source Type: research
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings
Gene Therapy, Published online: 25 April 2023; doi:10.1038/s41434-023-00401-5Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings (Source: Gene Therapy)
Source: Gene Therapy - April 25, 2023 Category: Genetics & Stem Cells Authors: Deepa H. Chand Rui Sun Karim A. Diab Damien Kenny Francis Fonyuy Tukov Source Type: research
rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality
Gene Therapy, Published online: 19 April 2023; doi:10.1038/s41434-023-00400-6rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality (Source: Gene Therapy)
Source: Gene Therapy - April 19, 2023 Category: Genetics & Stem Cells Authors: Seyedeh Zeinab Mirjalili Mohanna Andrea J. Korecki Elizabeth M. Simpson Source Type: research
Combinatorial gene therapy for epilepsy: Gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures
Gene Therapy, Published online: 07 April 2023; doi:10.1038/s41434-023-00399-wCombinatorial gene therapy for epilepsy: Gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures (Source: Gene Therapy)
Source: Gene Therapy - April 7, 2023 Category: Genetics & Stem Cells Authors: Esbj örn Melin My Andersson Casper R. G øtzsche Jenny Wickham Yuzhe Huang Julia Alicja Szczygiel Arnie Boender S øren H. Christiansen Lars Pinborg David P. D. Woldbye Merab Kokaia Source Type: research
Large-scale purification of functional AAV particles packaging the full genome using short-term ultracentrifugation with a zonal rotor
Gene Therapy, Published online: 28 March 2023; doi:10.1038/s41434-023-00398-xLarge-scale purification of functional AAV particles packaging the full genome using short-term ultracentrifugation with a zonal rotor (Source: Gene Therapy)
Source: Gene Therapy - March 28, 2023 Category: Genetics & Stem Cells Authors: Mikako Wada Naoya Uchida Guillermo Posadas-Herrera Hiromi Hayashita-Kinoh Yuji Tsunekawa Yukihiko Hirai Takashi Okada Source Type: research
