Results 21 to 30 of about 11,459,182 (339)
Molecular Therapy: Methods & Clinical Development, 2016 Recombinant adeno-associated viral (rAAV) vectors containing oversized genomes provide transgene expression despite low efficiency packaging of complete genomes.
Sirkka Kyostio-Moore +9 more
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Exosome-mediated stable epigenetic repression of HIV-1
Nature Communications, 2021 A strategy to control HIV-1 infection is to stably repress HIV-1 and induce “deep latency”. Here the authors show that a recombinant anti-HIV-1-1 protein can be packaged as mRNA into exosomes and delivered systemically to repress HIV-1-1 within the ...
Surya Shrivastava +7 more
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CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future
Frontiers in Oncology, 2020 A series of recent discoveries harnessing the adaptive immune system of prokaryotes to perform targeted genome editing is having a transformative influence across the biological sciences. The discovery of Clustered Regularly Interspaced Short Palindromic
F. Uddin, C. Rudin, T. Sen
semanticscholar +1 more source
Gut, 1982 Gene therapy is not yet possible, but may become feasible soon, particularly for well understood gene defects. Although treatment of a patient raises no ethical problems once it can be done well, changing the genes of an early embryo is more difficult, controversial and unlikely to be required clinically.
openaire +4 more sources
Molecular Metabolism, 2017 Objective: Type 1 diabetes is characterized by autoimmune destruction of β-cells leading to severe insulin deficiency. Although many improvements have been made in recent years, exogenous insulin therapy is still imperfect; new therapeutic approaches ...
Cristina Mallol +10 more
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Scientific Reports, 2022 As photoreceptor cells die during retinal degeneration, the surrounding microenvironment undergoes significant changes that are increasingly recognized to play a prominent role in determining the efficacy of therapeutic interventions.
A. Matsuyama +4 more
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Antibody-based CCR5 blockade protects Macaques from mucosal SHIV transmission
Nature Communications, 2021 CCR5 is a co-receptor for many transmitted HIV strains. Here, the authors show that biweekly injection of the CCR5-specific antibody Leronlimab protects rhesus macaques against infection following repeated intrarectal challenges of a CCR5-tropic SHIV.
Xiao L. Chang +32 more
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Nature Communications, 2020 Adeno-associated viruses (AAVs) are vehicles for gene therapy in humans, but currently only a limited amount of AAV serotypes is available. Here, the authors identify a novel AAV, AAVv66, and demonstrate enhanced production yields, virion stability, and ...
Hung-Lun Hsu +14 more
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The once and future gene therapy
Nature Communications, 2020 Gene therapy is at an inflection point. Recent successes in genetic medicine have paved the path for a broader second wave of therapies and laid the foundation for next-generation technologies. This comment summarizes recent advances and expectations for
Karen Bulaklak, C. Gersbach
semanticscholar +1 more source
Hematology/Oncology Clinics of North America, 2017 Gene therapy refers to a rapidly growing field of medicine in which genes are introduced into the body to treat or prevent diseases. Although a variety of methods can be used to deliver the genetic materials into the target cells and tissues, modified viral vectors represent one of the more common delivery routes because of its transduction efficiency ...
Daniel E, Bauer, Donald B, Kohn
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