Logo do repositório
 
Publicação

Host Cell Restriction Factors Blocking Efficient Vector Transduction

2023-03Recensão Acesso aberto
Ver registo simples
dc.contributor.authorCoroadinha, Ana Sofia
dc.contributor.institutionInstituto de Tecnologia Química e Biológica António Xavier (ITQB)
dc.contributor.pblMDPI - Multidisciplinary Digital Publishing Institute
dc.date.accessioned2023-09-30T22:21:33Z
dc.date.available2023-09-30T22:21:33Z
dc.date.issued2023-03
dc.descriptionPublisher Copyright: © 2023 by the author.
dc.description.abstractGene therapy relies on the delivery of genetic material to the patient’s cells in order to provide a therapeutic treatment. Two of the currently most used and efficient delivery systems are the lentiviral (LV) and adeno-associated virus (AAV) vectors. Gene therapy vectors must successfully attach, enter uncoated, and escape host restriction factors (RFs), before reaching the nucleus and effectively deliver the therapeutic genetic instructions to the cell. Some of these RFs are ubiquitously expressed in mammalian cells, while others are cell-specific, and others still are expressed only upon induction by danger signals as type I interferons. Cell restriction factors have evolved to protect the organism against infectious diseases and tissue damage. These restriction factors can be intrinsic, directly acting on the vector, or related with the innate immune response system, acting indirectly through the induction of interferons, but both are intertwined. The innate immunity is the first line of defense against pathogens and, as such cells derived from myeloid progenitors (but not only), are well equipped with RFs to detect pathogen-associated molecular patterns (PAMPs). In addition, some non-professional cells, such as epithelial cells, endothelial cells, and fibroblasts, play major roles in pathogen recognition. Unsurprisingly, foreign DNA and RNA molecules are among the most detected PAMPs. Here, we review and discuss identified RFs that block LV and AAV vector transduction, hindering their therapeutic efficacy.en
dc.description.versionpublishersversion
dc.description.versionpublished
dc.format.extent1672286
dc.identifier.doi10.3390/cells12050732
dc.identifier.issn2073-4409
dc.identifier.otherPURE: 72625596
dc.identifier.otherPURE UUID: 65ee9ba4-17c4-4ba8-9d77-1c8127b5fad3
dc.identifier.otherScopus: 85149757483
dc.identifier.otherPubMed: 36899868
dc.identifier.urihttp://hdl.handle.net/10362/158544
dc.identifier.urlhttps://www.scopus.com/pages/publications/85149757483
dc.language.isoeng
dc.peerreviewedyes
dc.subjectadeno-associated virus vectors
dc.subjectgene therapy
dc.subjecthost restriction factors
dc.subjectinnate-immune response
dc.subjectlentiviral vectors
dc.subjecttransduction
dc.subjectGeneral Biochemistry,Genetics and Molecular Biology
dc.subjectSDG 3 - Good Health and Well-being
dc.titleHost Cell Restriction Factors Blocking Efficient Vector Transductionen
dc.title.subtitleChallenges in Lentiviral and Adeno-Associated Vector Based Gene Therapiesen
dc.typereview
degois.publication.issue5
degois.publication.titleCells
degois.publication.volume12
dspace.entity.typePublication
rcaap.rightsopenAccess

Ficheiros

Principais
A mostrar 1 - 1 de 1
Miniatura
Nome:
Host_Cell_Restriction_Factors_Blocking_Efficient_Vector_Transduction_Challenges_in_Lentiviral_and_Adeno_Associated_Vector_Based_Gene_Therapies.pdf
Tamanho:
1.59 MB
Formato:
Adobe Portable Document Format
Ver/Abrir

Coleções

Home collection (ITQB)