A short hepatitis C virus NS5A peptide expression by AAV vector modulates human T cell activation and reduces vector immunogenicity
Gene Therapy, Published online: 11 November 2021; doi:10.1038/s41434-021-00302-5A short hepatitis C virus NS5A peptide expression by AAV vector modulates human T cell activation and reduces vector immunogenicity (Source: Gene Therapy)
Source: Gene Therapy - November 11, 2021 Category: Genetics & Stem Cells Authors: Winston Colon-Moran Alan Baer Gauri Lamture Jack T. Stapleton Joseph W. Fischer Nirjal Bhattarai Source Type: research
Progress on siRNA-based gene therapy targeting secondary injury after intracerebral hemorrhage
Gene Therapy, Published online: 10 November 2021; doi:10.1038/s41434-021-00304-3Progress on siRNA-based gene therapy targeting secondary injury after intracerebral hemorrhage (Source: Gene Therapy)
Source: Gene Therapy - November 10, 2021 Category: Genetics & Stem Cells Authors: Daniyah A. Almarghalani Zahoor A. Shah Source Type: research
Endotoxin contamination in commercially available Cas9 proteins potentially induces T-cell mediated responses
Gene Therapy, Published online: 08 November 2021; doi:10.1038/s41434-021-00301-6Endotoxin contamination in commercially available Cas9 proteins potentially induces T-cell mediated responses (Source: Gene Therapy)
Source: Gene Therapy - November 8, 2021 Category: Genetics & Stem Cells Authors: Vijaya L. Simhadri Joseph R. McGill Zuben E. Sauna Source Type: research
Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy
Gene Therapy, Published online: 05 November 2021; doi:10.1038/s41434-021-00300-7Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy (Source: Gene Therapy)
Source: Gene Therapy - November 5, 2021 Category: Genetics & Stem Cells Authors: Vahid Farrokhi Jason Walsh Joe Palandra Joanne Brodfuehrer Teresa Caiazzo Jane Owens Michael Binks Srividya Neelakantan Florence Yong Pinky Dua Caroline Le Guiner Hendrik Neubert Source Type: research
Consideration with “Intratumoral gene therapy versus intravenous gene therapy for distant metastasis control with DDMC non-viral vector–p53”
Gene Therapy, Published online: 12 October 2021; doi:10.1038/s41434-021-00298-yConsideration with “Intratumoral gene therapy versus intravenous gene therapy for distant metastasis control with DDMC non-viral vector–p53” (Source: Gene Therapy)
Source: Gene Therapy - October 12, 2021 Category: Genetics & Stem Cells Authors: Y. Onishi P. Zarogoulidis R-C Ji M. Onishi N. Kubota Y. Eshita Source Type: research
A PCR-amplified transgene fragment flanked by a single copy of a truncated inverted terminal repeat for recombinant adeno-associated virus production prevents unnecessary plasmid DNA packaging
Gene Therapy, Published online: 11 October 2021; doi:10.1038/s41434-021-00299-xA PCR-amplified transgene fragment flanked by a single copy of a truncated inverted terminal repeat for recombinant adeno-associated virus production prevents unnecessary plasmid DNA packaging (Source: Gene Therapy)
Source: Gene Therapy - October 11, 2021 Category: Genetics & Stem Cells Authors: Kumi Adachi Taro Tomono Hironori Okada Yusuke Shiozawa Motoko Yamamoto Yoshitaka Miyagawa Takashi Okada Source Type: research
Rational engineering of a functional CpG-free ITR for AAV gene therapy
Gene Therapy, Published online: 06 October 2021; doi:10.1038/s41434-021-00296-0Rational engineering of a functional CpG-free ITR for AAV gene therapy (Source: Gene Therapy)
Source: Gene Therapy - October 6, 2021 Category: Genetics & Stem Cells Authors: Xiufang Pan Yongping Yue Maria Boftsi Lakmini P. Wasala Ngoc Tam Tran Keqing Zhang David J. Pintel Phillip W. L. Tai Dongsheng Duan Source Type: research
Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models
Gene Therapy, Published online: 06 October 2021; doi:10.1038/s41434-021-00292-4Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models (Source: Gene Therapy)
Source: Gene Therapy - October 6, 2021 Category: Genetics & Stem Cells Authors: Ellie M. Chilcott Evalyne W. Muiruri Theodore C. Hirst Rafael J. Y áñez-Muñoz Source Type: research
Oncolytic Newcastle disease virus expressing the co-stimulator OX40L as immunopotentiator for colorectal cancer therapy
Gene Therapy, Published online: 04 October 2021; doi:10.1038/s41434-021-00256-8Oncolytic Newcastle disease virus expressing the co-stimulator OX40L as immunopotentiator for colorectal cancer therapy (Source: Gene Therapy)
Source: Gene Therapy - October 4, 2021 Category: Genetics & Stem Cells Authors: Limin Tian Tianyan Liu Shan Jiang Yukai Cao Kai Kang Han Su Guiping Ren Zhenzhong Wang Wei Xiao Deshan Li Source Type: research
Correction of a knock-in mouse model of acrodysostosis with gene therapy using a rAAV9-CAG-human PRKAR1A vector
Gene Therapy, Published online: 01 October 2021; doi:10.1038/s41434-021-00286-2Correction of a knock-in mouse model of acrodysostosis with gene therapy using a rAAV9-CAG-human PRKAR1A vector (Source: Gene Therapy)
Source: Gene Therapy - October 1, 2021 Category: Genetics & Stem Cells Authors: Yasemin Özgür-Günes Catherine Le Stunff Malha Chedik Marie-Pierre Belot Pierre-Hadrien Becker V éronique Blouin Pierre Bougn ères Source Type: research
CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing
Gene Therapy, Published online: 01 October 2021; doi:10.1038/s41434-021-00297-zCRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing (Source: Gene Therapy)
Source: Gene Therapy - October 1, 2021 Category: Genetics & Stem Cells Authors: Pouir é Yaméogo Benjamin L. Duch êne Nathalie Majeau Jacques P. Tremblay Source Type: research
Long-term safety and efficacy of intramyocardial adenovirus-mediated VEGF-DΔNΔC gene therapy eight-year follow-up of phase I KAT301 study
Gene Therapy, Published online: 01 October 2021; doi:10.1038/s41434-021-00295-1Long-term safety and efficacy of intramyocardial adenovirus-mediated VEGF-DΔNΔC gene therapy eight-year follow-up of phase I KAT301 study (Source: Gene Therapy)
Source: Gene Therapy - October 1, 2021 Category: Genetics & Stem Cells Authors: Aleksi J. Leikas Iiro Hassinen Antti Hedman Antti Kivel ä Seppo Yl ä-Herttuala Juha E. K. Hartikainen Source Type: research
Correction: Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)
Gene Therapy, Published online: 29 September 2021; doi:10.1038/s41434-021-00293-3Correction: Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI) (Source: Gene Therapy)
Source: Gene Therapy - September 29, 2021 Category: Genetics & Stem Cells Authors: Jennifer E. Adair Lindsay Androski Lois Bayigga Deus Bazira Eugene Brandon Lynda Dee Steven Deeks Mohamed Draz Karine Dub é Mark Dybul Umut Gurkan Evelyn Harlow Cissy Kityo Michael Louella Punam Malik Vikram Mathews Adrian McKemey Henry Mugerwa Daniel Mu Source Type: research
Injection pressure levels for creating blebs during subretinal gene therapy
Gene Therapy, Published online: 28 September 2021; doi:10.1038/s41434-021-00294-2Injection pressure levels for creating blebs during subretinal gene therapy (Source: Gene Therapy)
Source: Gene Therapy - September 28, 2021 Category: Genetics & Stem Cells Authors: Brittni A. Scruggs Huber Martins Vasconcelos Jr. Mariana Matioli da Palma Katie Kogachi Mark E. Pennesi Paul Yang Steven T. Bailey Andreas K. Lauer Source Type: research
Rs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation
Gene Therapy, Published online: 22 September 2021; doi:10.1038/s41434-021-00290-6Rs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation (Source: Gene Therapy)
Source: Gene Therapy - September 22, 2021 Category: Genetics & Stem Cells Authors: Yong Zeng Haohua Qian Maria Mercedes Campos Yichao Li Camasamudram Vijayasarathy Paul A. Sieving Source Type: research
