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How vectors work to transfer genetic material. Gene therapy utilizes the delivery of DNA into cells, which can be accomplished by several methods, summarized below. The two major classes of methods are those that use recombinant viruses (sometimes called biological nanoparticles or viral vectors) and those that use naked DNA or DNA complexes (non-viral methods).
The adenoviral capsid, an icosahedron, features a fibre "knob" at each of its 12 vertices. These fibre proteins mediate cell entry—greatly affecting efficacy and contribute to its broad tropism—notably via coxsackie–adenovirus receptors (CARs). [34] [37] Adenoviral vectors can induce robust innate and adaptive immune responses. [38]
Several features make AAV an attractive candidate for creating viral vectors for gene therapy, and for the creation of isogenic human disease models. [3] Gene therapy vectors using AAV can infect both dividing and quiescent cells and persist in an extrachromosomal state without integrating into the genome of the host cell.
In gene therapy a gene that is intended for delivery is packaged into a replication-deficient viral particle to form a viral vector. [29] Viruses used for gene therapy to date include retrovirus, adenovirus, adeno-associated virus and herpes simplex virus. However, there are drawbacks to using viruses to deliver genes into cells.
The original issue focused on viral vectors for gene delivery vectors and use of gene therapy treatments for oncology. Current Gene Therapy was among the first gene therapy focused journals to publish on the use of Alphaviruses for gene therapy, [ 2 ] oncolytic virotherapy using adenoviral -mediated gene transfer, [ 3 ] and the use of Fas ...
Examples of mammalian expression vectors include the adenoviral vectors, [38] the pSV and the pCMV series of plasmid vectors, vaccinia and retroviral vectors, [39] as well as baculovirus. [30] The promoters for cytomegalovirus (CMV) and SV40 are commonly used in mammalian expression vectors to drive gene expression. Non-viral promoter, such as ...
Since HEK 293 cells express a number of adenoviral genes, they can be used to propagate adenoviral vectors in which these genes (typically, E1 and E3) are deleted, such as AdEasy. [24] However, homologous recombination between the inserted cellular Ad5 sequence and the vector sequence, although rare, can restore the replication capacity to the ...
Virotherapy is a treatment using biotechnology to convert viruses into therapeutic agents by reprogramming viruses to treat diseases. There are three main branches of virotherapy: anti-cancer oncolytic viruses, viral vectors for gene therapy and viral immunotherapy.
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