Search results - 17 results
Collaborative data collection by TREAT-NMD Registries to support post-marketing surveillance in Spinal Muscular Atrophy
atrophy (SMA) is a rare genetic neuromuscular disorder characterized by a loss of motor neurons in the spinal cord and the brainstem, leading to muscle weakness and atrophy. The estimated incidence is ...
Engineering nucleases for gene targeting: safety and regulatory considerations.
(2014) Keywords: Deoxyribonucleases Gene Targeting Genetic Therapy Humans SAFETY Abstract: Nuclease-based gene targeting (NBGT) represents a significant breakthrough in targeted genome editing since it is ...
Biosafety of viral vectors commonly used in gene therapy and vaccination.
Publication Type: Peer reviewed scientific article Authors: Philippe Herman Source: Curr Gene Ther, Volume 13, Issue 6, p.383-4 (2013) Keywords: Genetic Therapy Genetic Vectors Hazardous Substances ...
Environmental risk assessment of clinical trials involving modified vaccinia virus Ankara (MVA)-based vectors.
Animals Clinical Trials as Topic Genetic Therapy Genetic Vectors Hazardous Substances Humans Risk Assessment Vaccination vaccinia virus Viral Vaccines Abstract: The modified vaccinia virus Ankara (MVA) ...
General considerations on the biosafety of virus-derived vectors used in gene therapy and vaccination.
p.385-94 (2013) Keywords: Animals Clinical Trials as Topic Genetic Therapy Genetic Vectors Hazardous Substances Humans Organisms, Genetically Modified Risk Assessment Vaccination Viruses Abstract: This ...
The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia
POPULATION prevalence protein Rare disease REGISTRIES Registry successful Therapy UK utilization Abstract: Duchenne muscular dystrophy (DMD) is an X-linked genetic disease, caused by the absence of the ...
Intra-host variation structure of classical swine fever virus NS5B in relation to antiviral therapy.
Substitution Animals Antiviral Agents Classical Swine Fever Classical swine fever virus Enzyme Inhibitors Genetic Variation Imidazoles Pyridines RNA Replicase Swine Viral Proteins Abstract: Classical swine fever ...
Biosafety aspects of modified vaccinia virus Ankara (MVA)-based vectors used for gene therapy or vaccination.
Clinical Trials as Topic Gene Transfer Techniques Genetic Therapy Genetic Vectors Humans Mammals Neoplasms Poxviridae Infections Practice Guidelines as Topic Vaccination vaccinia virus Viral Vaccines ...
Contained use of bacteriophages: Risk assessment and biosafety recommendations
antibodies application applications AS assessment bacteria Bacteriophages biosafety Biotechnology containment Control delivery Dna DNA vaccines environment Evaluating Evolution gene gene therapy gene transfer ...
Contained use of Bacteriophages: Risk assessment and biosafety recommendations2463
environment epub Evaluating Evolution factors Field gene gene therapy gene transfer general Genetic Genome genomic health Human human health implement Laboratories lysis management measure measures medical ...