Results 21 to 30 of about 1,204,526 (212)
Functional roles of the membrane-associated AAV protein MAAP
Scientific Reports, 2021 With a limited coding capacity of 4.7 kb, adeno-associated virus (AAV) genome has evolved over-lapping genes to maximise the usage of its genome. An example is the recently found ORF in the cap gene, encoding membrane-associated accessory protein (MAAP),
Lionel Galibert +13 more
doaj +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
Cold Spring Harbor Perspectives in Medicine, 2012 Over 450 million people worldwide suffer from hearing loss, leading to an estimated economic burden of ∼$750 billion. The past decade has seen significant advances in the understanding of the molecular mechanisms that contribute to hearing, and the environmental and genetic factors that can go awry and lead to hearing loss.
Lawrence R, Lustig, Omar, Akil
openaire +4 more sources
Biology of Blood and Marrow Transplantation, 1999 For the past two decades, concerted efforts have been made to treat human disease by replacing nonfunctioning genes in cells or by correcting mutations that produce disease. Successful application of these methods could lead to effective therapies for a variety of genetic and acquired diseases, many of which are not treatable today.
openaire +2 more sources
Gene therapy for arthritis [PDF]
Expert Opinion on Investigational Drugs, 1994 In the two years since arthritis gene therapy was last reviewed in this journal, there has been rapid progress on several fronts. Although vector development remains a slow process and long-term gene expression is not easily obtained, very encouraging preclinical data in animal models of arthritis are now emerging.
C H, Evans, P D, Robbins
openaire +2 more sources
Scientific Reports, 2023 Process development for transferring lab-scale research workflows to automated manufacturing procedures is critical for chimeric antigen receptor (CAR)-T cell therapies.
Nadine von Auw +10 more
doaj +1 more source
Oral Diseases, 2013 Applications of gene therapy have been evaluated in virtually every oral tissue, and many of these have proved successful at least in animal models. While gene therapy will not be used routinely in the next decade, practitioners of oral medicine should be aware of the potential of this novel type of treatment that doubtless will benefit many patients ...
openaire +2 more sources
Macrophage Transcriptomic Alterations Driven by Alphavirus-Based Cancer Immunotherapy Vectors
Journal of Immunology ResearchCancer cells promote the polarisation of tumour-associated macrophages (TAMs) into pro-tumorigenic M2-like phenotype, contributing to cancer progression. Reprogramming TAMs by viral immunotherapy vectors represents a promising strategy for cancer therapy.
Ksenija Korotkaja +3 more
doaj +1 more source
EMBO Molecular MedicinePhysiological regulation of transgene expression is a major challenge in gene therapy. Onasemnogene abeparvovec (Zolgensma®) is an approved adeno-associated virus (AAV) vector gene therapy for infants with spinal muscular atrophy (SMA), however, adverse ...
Qing Xie +16 more
doaj +1 more source
Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes
Molecular Therapy: Methods & Clinical Development, 2018 The generation of clinical good manufacturing practices (GMP)-grade adeno-associated virus (AAV) vectors requires purification strategies that support the generation of vectors of high purity, and that exhibit a good safety and efficacy profile. To date,
Shelley A. Nass +9 more
doaj +1 more source