Results 1 to 10 of about 3,726,105 (294)
A gene drive is a gene drive: the debate over lumping or splitting definitions. [PDF]
Nat Commun, 2023 We address a controversy over use of the term “gene drive” to include both natural and synthetic genetic elements that promote their own transmission within a population, arguing that this broad definition is both practical and has advantages for risk analysis.
James SL +3 more
europepmc +6 more sources
The challenge of measuring mosquito flight performance: going beyond sterile insect technique and into transgenic and gene drive-based approaches [PDF]
Open BiologyInvasive insects inflict global costs of more than 70 billion USD annually by destroying crops and spreading disease-causing pathogens. Sterile insect technique (SIT), an insect population control method, involves the irradiation or chemical ...
Paola Najera +4 more
doaj +2 more sources
Driving down malaria transmission with engineered gene drives
Frontiers in Genetics, 2022 The last century has witnessed the introduction, establishment and expansion of mosquito-borne diseases into diverse new geographic ranges. Malaria is transmitted by female Anopheles mosquitoes.
William T. Garrood +5 more
doaj +4 more sources
Assessing target genes for homing suppression gene drive. [PDF]
EMBO JAbstract Gene drives are engineered alleles that bias their own inheritance in offspring, enabling the spread of specific traits throughout a population. Targeting female fertility genes in a gene drive can be an efficient strategy for population suppression.
Xu X +7 more
europepmc +3 more sources
Gene drives as a response to infection and resistance
Infection and Drug Resistance, 2019 Tuna C Hayirli,1 Peter F Martelli2 1Harvard Medical School, Boston, MA, USA; 2Department of Healthcare Administration, Sawyer Business School, Suffolk University, Boston, MA, USA Abstract: Vector-borne infectious diseases continue to be a major threat to
Hayirli TC, Martelli PF
doaj +5 more sources
Molecular Therapy: Methods & Clinical Development, 2022 Duchenne muscular dystrophy (DMD) is typically caused by mutations that disrupt the DMD reading frame, but nonsense mutations in the 5′ part of the gene induce utilization of an internal ribosomal entry site (IRES) in exon 5, driving expression of a ...
Nicolas Wein +12 more
doaj +1 more source
Molecular Therapy: Methods & Clinical Development, 2021 The increasing demand for adeno-associated virus (AAV) vectors, a result from the surging interest for their potential to cure human genetic diseases by gene transfer, tumbled on low-performing production systems. Innovative improvements to increase both
Chenghui Yu +8 more
doaj +1 more source
Molecular Therapy: Nucleic Acids, 2021 Facioscapulohumeral muscular dystrophy (FSHD) arises from epigenetic changes that de-repress the DUX4 gene in muscle. The full-length DUX4 protein causes cell death and muscle toxicity, and therefore we hypothesize that FSHD therapies should center on ...
Afrooz Rashnonejad +4 more
doaj +1 more source
Molecular Therapy: Methods & Clinical Development, 2022 In a phase 1/2, open-label dose escalation trial, we delivered rAAVrh74.MCK.GALGT2 (also B4GALNT2) bilaterally to the legs of two boys with Duchenne muscular dystrophy using intravascular limb infusion.
Kevin M. Flanigan +14 more
doaj +1 more source
Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1
Molecular Therapy: Methods & Clinical Development, 2021 Limb girdle muscular dystrophy (LGMD) 2A/R1, caused by mutations in the CAPN3 gene and CAPN3 loss of function, is known to play a role in disease pathogenicity.
Zarife Sahenk +8 more
doaj +1 more source