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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
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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
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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
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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
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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
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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
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Frontiers in Bioengineering and Biotechnology, 2023 Gene drive-modified mosquitoes (GDMMs) are proposed as new tools for control and elimination of malaria and other mosquito-borne diseases, and promising results have been observed from testing conducted in containment. Although still at an early stage of
Stephanie L. James +3 more
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Selfing is the safest sex for Caenorhabditis tropicalis
eLife, 2021 Mating systems have profound effects on genetic diversity and compatibility. The convergent evolution of self-fertilization in three Caenorhabditis species provides a powerful lens to examine causes and consequences of mating system transitions.
Luke M Noble +13 more
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Anopheles gambiae Genome Conservation as a Resource for Rational Gene Drive Target Site Selection
Insects, 2021 The increase in molecular tools for the genetic engineering of insect pests and disease vectors, such as Anopheles mosquitoes that transmit malaria, has led to an unprecedented investigation of the genomic landscape of these organisms.
Nace Kranjc +3 more
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Molecular Therapy: Methods & Clinical Development, 2019 rAAVrh74.MCK.GALGT2 is a surrogate gene therapy that inhibits muscular dystrophy in multiple animal models. Here, we report on a dose-response study of functional muscle GALGT2 expression as well as toxicity and biodistribution studies after systemic ...
Deborah A. Zygmunt +10 more
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