Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic
出版年份 2023 全文链接
标题
Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic
作者
关键词
-
出版物
GENE THERAPY
Volume -, Issue -, Pages -
出版商
Springer Science and Business Media LLC
发表日期
2023-08-23
DOI
10.1038/s41434-023-00417-x
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Why gene therapies must go virus-free
- (2023) Cormac Sheridan NATURE BIOTECHNOLOGY
- Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution
- (2023) Marti Cabanes-Creus et al. Molecular Therapy-Methods & Clinical Development
- Importance of non-human primates as model system for gene therapy development in ophthalmology
- (2022) Fabian Wozar et al. KLINISCHE MONATSBLATTER FUR AUGENHEILKUNDE
- Gene Therapy Advances: A Meta-Analysis of AAV Usage in Clinical Settings
- (2022) Hau Kiu Edna Au et al. Frontiers in Medicine
- Addressing high dose AAV toxicity – ‘one and done’ or ‘slower and lower’?
- (2022) Takashi Kei Kishimoto et al. EXPERT OPINION ON BIOLOGICAL THERAPY
- A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders
- (2022) Francesca Tucci et al. Nature Communications
- Immunogenicity and toxicity of AAV gene therapy
- (2022) Hildegund C. J. Ertl Frontiers in Immunology
- Optimized AAV Vectors for TMC1 Gene Therapy in a Humanized Mouse Model of DFNB7/11
- (2022) Irina Marcovich et al. Biomolecules
- The clinical landscape for AAV gene therapies
- (2021) Dmitry A. Kuzmin et al. NATURE REVIEWS DRUG DISCOVERY
- Evolving AAV-delivered therapeutics towards ultimate cures
- (2021) Xiangjun He et al. JOURNAL OF MOLECULAR MEDICINE-JMM
- CAR-T cell therapy: current limitations and potential strategies
- (2021) Robert C. Sterner et al. Blood Cancer Journal
- T Cell-Mediated Immune Responses to AAV and AAV Vectors
- (2021) Hildegund C. J. Ertl Frontiers in Immunology
- Repeated systemic dosing of AAV vectors in immunocompetent mice after blockade of T-cell costimulatory pathways
- (2021) Chen Zhong et al. HUMAN GENE THERAPY
- Huntingtin and the Synapse
- (2021) Jessica C. Barron et al. Frontiers in Cellular Neuroscience
- Precise CAG repeat contraction in a Huntington’s Disease mouse model is enabled by gene editing with SpCas9-NG
- (2021) Seiya Oura et al. Communications Biology
- Fourth Boy Dies in Clinical Trial of Astellas' AT132
- (2021) Alex Philippidis HUMAN GENE THERAPY
- Species selection for nonclinical safety assessment of drug candidates: Examples of current industry practice
- (2021) Rostam Namdari et al. REGULATORY TOXICOLOGY AND PHARMACOLOGY
- Utility of microminipigs for evaluating liver-mediated gene expression in the presence of neutralizing antibody against vector capsid
- (2020) Ryota Watano et al. GENE THERAPY
- AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer
- (2020) Helena Costa Verdera et al. MOLECULAR THERAPY
- Focus shifts to antibody cocktails for COVID-19 cytokine storm
- (2020) Charlotte Harrison NATURE BIOTECHNOLOGY
- Towards Clinical Implementation of Adeno-Associated Virus (AAV) Vectors for Cancer Gene Therapy: Current Status and Future Perspectives
- (2020) Ulrich T. Hacker et al. Cancers
- Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology
- (2020) Juliette Hordeaux et al. HUMAN GENE THERAPY
- After Third Death, Audentes' AT132 Remains on Clinical Hold
- (2020) Alex Philippidis HUMAN GENE THERAPY
- Huntington disease: new insights into molecular pathogenesis and therapeutic opportunities
- (2020) Sarah J. Tabrizi et al. Nature Reviews Neurology
- Advances in selecting appropriate non-rodent species for regulatory toxicology research: Policy, ethical, and experimental considerations
- (2020) Yong-Wook Son et al. REGULATORY TOXICOLOGY AND PHARMACOLOGY
- Gene therapy for monogenic inherited disorders—opportunities and challenges
- (2020) Janbernd Kirschner et al. Deutsches Arzteblatt International
- Current Clinical Applications of In Vivo Gene Therapy with AAVs
- (2020) Jerry R. Mendell et al. MOLECULAR THERAPY
- The once and future gene therapy
- (2020) Karen Bulaklak et al. Nature Communications
- Predictivity/Translatability of Toxicities Observed in Nonclinical Toxicology Studies to Clinical Safety Outcomes in Drug Development: Case Examples
- (2019) Nicola J. Stagg et al. INTERNATIONAL JOURNAL OF TOXICOLOGY
- Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
- (2018) Christian Hinderer et al. HUMAN GENE THERAPY
- AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model
- (2018) Melvin M. Evers et al. MOLECULAR THERAPY
- Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility
- (2018) Jayesh A. Kulkarni et al. Nucleic Acid Therapeutics
- Emerging Issues in AAV-Mediated In Vivo Gene Therapy
- (2018) Pasqualina Colella et al. Molecular Therapy-Methods & Clinical Development
- Huntington's disease: a clinical review
- (2017) P. McColgan et al. EUROPEAN JOURNAL OF NEUROLOGY
- Lipid Nanoparticle Systems for Enabling Gene Therapies
- (2017) Pieter R. Cullis et al. MOLECULAR THERAPY
- After Glybera's withdrawal, what's next for gene therapy?
- (2017) Melanie Senior NATURE BIOTECHNOLOGY
- Next-generation AAV vectors for clinical use: an ever-accelerating race
- (2017) Jonas Weinmann et al. VIRUS GENES
- Persistent Expression of Dopamine-Synthesizing Enzymes 15 Years After Gene Transfer in a Primate Model of Parkinson's Disease
- (2017) Yoshihide Sehara et al. Human Gene Therapy Clinical Development
- Current Progress in Therapeutic Gene Editing for Monogenic Diseases
- (2016) Versha Prakash et al. MOLECULAR THERAPY
- Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?
- (2016) Randy J Chandler et al. MOLECULAR THERAPY
- In vivo tissue-tropism of adeno-associated viral vectors
- (2016) Arun Srivastava Current Opinion in Virology
- E Pluribus Unum: 50 Years of Research, Millions of Viruses, and One Goal—Tailored Acceleration of AAV Evolution
- (2015) Dirk Grimm et al. MOLECULAR THERAPY
- Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy
- (2014) Balaji Balakrishnan et al. CURRENT GENE THERAPY
- Gene Therapies for Cancer: Strategies, Challenges and Successes
- (2014) Swadesh K. Das et al. JOURNAL OF CELLULAR PHYSIOLOGY
- Engineering adeno-associated viruses for clinical gene therapy
- (2014) Melissa A. Kotterman et al. NATURE REVIEWS GENETICS
- Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse
- (2014) Daniel J. Schuster et al. Frontiers in Neuroanatomy
- Treatment of Diabetes and Long-Term Survival After Insulin and Glucokinase Gene Therapy
- (2013) D. Callejas et al. DIABETES
- The immune privilege of testis and gravid uterus: Same difference?
- (2013) Petra Arck et al. MOLECULAR AND CELLULAR ENDOCRINOLOGY
- Pre-existing Anti–Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy
- (2013) Vedell Louis Jeune et al. Human Gene Therapy Methods
- Lessons Learned from the Clinical Development and Market Authorization of Glybera
- (2013) Laura M. Bryant et al. Human Gene Therapy Clinical Development
- Directed evolution of novel adeno-associated viruses for therapeutic gene delivery
- (2012) M A Bartel et al. GENE THERAPY
- Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial
- (2012) D Gaudet et al. GENE THERAPY
- The Minipig as Nonrodent Species in Toxicology—Where Are We Now?
- (2012) Niels Christian Ganderup et al. INTERNATIONAL JOURNAL OF TOXICOLOGY
- rAAV-Mediated Tumorigenesis: Still Unresolved After an AAV Assault
- (2012) Paul N Valdmanis et al. MOLECULAR THERAPY
- The privileged immunity of immune privileged organs: the case of the eye
- (2012) Inbal Benhar et al. Frontiers in Immunology
- Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents
- (2011) Roberto Calcedo et al. Clinical and Vaccine Immunology
- Considerations Regarding Nonhuman Primate Use in Safety Assessment of Biopharmaceuticals
- (2011) Lorrene A. Buckley et al. INTERNATIONAL JOURNAL OF TOXICOLOGY
- Enhancing the clinical potential of AAV vectors by capsid engineering to evade pre-existing immunity
- (2011) Melissa Bartel Frontiers in Microbiology
- Assessing the potential for AAV vector genotoxicity in a murine model
- (2010) H. Li et al. BLOOD
- Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors
- (2010) Sylvie Boutin et al. HUMAN GENE THERAPY
- Regulatory acceptability of the minipig in the development of pharmaceuticals, chemicals and other products
- (2010) Jan Willem van der Laan et al. JOURNAL OF PHARMACOLOGICAL AND TOXICOLOGICAL METHODS
- Host and Vector-dependent Effects on the Risk of Germline Transmission of AAV Vectors
- (2009) Patricia Favaro et al. MOLECULAR THERAPY
- Adeno-associated virus integration: virus versus vector
- (2008) R H Smith GENE THERAPY
Publish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn MoreAsk a Question. Answer a Question.
Quickly pose questions to the entire community. Debate answers and get clarity on the most important issues facing researchers.
Get Started