Gene Replacement by Homologous Recombination.
Sign Up to like & getrecommendations! Published in 2019 at "Methods in molecular biology"
DOI: 10.1007/978-1-4939-9210-2_8
Abstract: While homologous recombination-based gene replacement is about to be supplanted by more modern approaches, it is still retaining usefulness for genes that prove to be poor targets for CRISPR/cas-based approaches. Homologous recombination has proven to… read more here.
Keywords: homologous recombination; gene replacement; replacement homologous; recombination ... See more keywords
Feasibility of Using NF1-GRD and AAV for Gene Replacement Therapy in NF1-Associated Tumors
Sign Up to like & getrecommendations! Published in 2019 at "Gene therapy"
DOI: 10.1038/s41434-019-0080-9
Abstract: Neurofibromatosis type 1, including the highly aggressive malignant peripheral nerve sheath tumors (MPNSTs), is featured by the loss of functional neurofibromin 1 (NF1) protein resulting from genetic alterations. A major function of NF1 is suppressing… read more here.
Keywords: replacement therapy; grd; gene replacement; therapy nf1 ... See more keywords
[Gene Replacement Therapy for Inherited Retinal Dystrophies].
Sign Up to like & getrecommendations! Published in 2017 at "Klinische Monatsblatter fur Augenheilkunde"
DOI: 10.1055/s-0043-101817
Abstract: Characteristics of inherited retinal dystrophies include deficiencies in light perception and nervous conduction within the retina, leading to reduced vision or even blindness. In this context, the loss of function of photoreceptor-specific genes causes a… read more here.
Keywords: replacement therapy; retinal dystrophies; gene replacement; therapy inherited ... See more keywords
Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies
Sign Up to like & getrecommendations! Published in 2021 at "Expert Opinion on Investigational Drugs"
DOI: 10.1080/13543784.2021.1868434
Abstract: ABSTRACT Introduction Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused by mutations in the dystrophin (DMD) gene. Most patients die from respiratory failure or cardiomyopathy. There are significant unmet needs for treatments for… read more here.
Keywords: exon skipping; codon readthrough; gene replacement; stop codon ... See more keywords
Challenges and opportunities when transitioning from in vivo gene replacement to in vivo CRISPR/Cas9 therapies – a spotlight on hemophilia
Sign Up to like & getrecommendations! Published in 2022 at "Expert Opinion on Biological Therapy"
DOI: 10.1080/14712598.2022.2090241
Abstract: ABSTRACT Introduction Currently, a few in vivo gene replacement therapies are commercially available, with many in clinical development for the treatment of some inherited monogenic diseases. These disorders arise from mutations in genes encoding essential… read more here.
Keywords: gene replacement; replacement therapies; vivo gene; challenges opportunities ... See more keywords
Potential therapeutic targets for olfactory dysfunction in ciliopathies beyond single gene replacement.
Sign Up to like & getrecommendations! Published in 2021 at "Chemical senses"
DOI: 10.1093/chemse/bjab010
Abstract: Olfactory dysfunction is a common disorder in the general population. There are multiple causes, one of which being ciliopathies, an emerging class of human hereditary genetic disorders characterized by multiple symptoms due to defects in… read more here.
Keywords: olfactory dysfunction; gene replacement; dysfunction; therapeutic targets ... See more keywords
Gene replacement therapy after neuropathy onset provides therapeutic benefit in a model of CMT1X.
Sign Up to like & getrecommendations! Published in 2019 at "Human molecular genetics"
DOI: 10.1093/hmg/ddz199
Abstract: X-linked Charcot-Marie-Tooth disease (CMT1X), one of the commonest forms of inherited demyelinating neuropathy, results from GJB1 gene mutations causing loss of function of the gap junction protein connexin32 (Cx32). The aim of this study was… read more here.
Keywords: replacement therapy; gene replacement; model; gene ... See more keywords
LI-Detector: a Method for Curating Ordered Gene-Replacement Libraries
Sign Up to like & getrecommendations! Published in 2022 at "Microbiology Spectrum"
DOI: 10.1128/spectrum.00833-22
Abstract: The construction of ordered gene replacement libraries requires significant investment of time and resources to create a valuable community resource. During construction, technical errors may result in a limited number of incorrect mutants being made.… read more here.
Keywords: gene; gene replacement; replacement libraries; method ... See more keywords