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Vectors in gene therapy information


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).

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Vectors in gene therapy

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(FDA) in 2008. Concerns about the safety of adenovirus vectors were raised after the 1999 death of Jesse Gelsinger while participating in a gene therapy trial...

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Lentiviral vector in gene therapy

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Lentiviral vectors in gene therapy is a method by which genes can be inserted, modified, or deleted in organisms using lentiviruses. Lentiviruses are...

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Gene therapy

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DNA.: 5  As of 2017, such vectors were used in 20% of trials for gene therapy.: 10  Adenovirus vectors are mostly used in cancer treatments and novel...

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Viral vector

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Viral vectors are widely used in gene therapy due to their specific tropism and efficient transgene expression: as of 2022, all approved gene therapies were...

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Gene delivery

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a vector, which is designed to enter the desired host cell and deliver the transgene to that cell's genome. Vectors utilized as the method for gene delivery...

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Enzyme replacement therapy

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through the use of vectors, usually viral vectors. In gene therapy, a gene encoding for a certain protein is inserted into a vector. The vector containing the...

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Gene therapy for color blindness

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Gene therapy for color blindness is an experimental gene therapy of the human retina aiming to grant typical trichromatic color vision to individuals with...

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Gene therapy for osteoarthritis

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While viral vector gene therapies predominate, both viral and non-viral vectors have been developed as a means to deliver therapeutic genes. As the body...

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Retinal gene therapy using lentiviral vectors

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Gene therapy using lentiviral vectors was being explored in early stage trials as of 2009.[needs update] In a Phase I clinical trial of three patients...

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Etranacogene dezaparvovec

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Hemgenix is a gene therapy used for the treatment of hemophilia B. Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists...

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Designer baby

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germline gene therapy in treatment of disorders such as inherited mitochondrial diseases. Gene transfer to cells is usually by vector delivery. Vectors are...

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Suicide gene

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suicide gene by the desired fragment. Selection of vectors carrying the desired fragment is improved since vectors retaining the suicide gene result in cell...

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Retrovirus

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Laboratory. ISBN 978-0-87969-571-2. {Miller, A. D. (2006). Retroviral Vectors in Gene Therapy. Encyclopedia of Life Sciences. doi:10.1038/npg.els.0005741} Kurth...

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Virotherapy

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viral vectors for gene therapy and viral immunotherapy. These branches use three different types of treatment methods: gene overexpression, gene knockout...

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Valoctocogene roxaparvovec

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contain the gene for factor VIII, which is lacking in people with hemophilia A. It is an adeno-associated virus vector-based gene therapy. It is given...

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Current Gene Therapy

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gene expression and regulation, development of novel gene delivery vectors, and ex vivo gene therapies, animal models, and human therapeutic applications...

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Delandistrogene moxeparvovec

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of the dystrophin protein present in normal muscle cells. It is an adeno-associated virus vector-based gene therapy that is given by injection into a...

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Nadofaragene firadenovec

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a gene therapy for the treatment of bladder cancer. It is a non-replicating (cannot multiply in human cells) adenoviral vector-based gene therapy. The...

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Gene therapy for epilepsy

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Gene therapy is being studied for some forms of epilepsy. It relies on viral or non-viral vectors to deliver DNA or RNA to target brain areas where seizures...

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Expression vector

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another organism, and these vectors are called shuttle vectors. An expression vector must have elements necessary for gene expression. These may include...

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Genetically modified virus

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023. PMC 4477532. PMID 19446586. Lundstrom K (May 2018). "Viral Vectors in Gene Therapy". Diseases. 6 (2): 42. doi:10.3390/diseases6020042. PMC 6023384...

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Tumor suppressor gene

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alterations, viral methods of gene therapy for tumor suppressor genes have shown to be successful. In this method, vectors from viruses are used. The two...

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Molecular cloning

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disruption of essential genes within the patient's genome by insertional inactivation. In others, viral vectors used for gene therapy have been contaminated...

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Human genetic enhancement

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by means of a genetic modification. This could be done in order to cure diseases (gene therapy), prevent the possibility of getting a particular disease...

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Lipofectamine

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transfection was invented by Dr. Yongliang Chu. Lipofection Transfection Vectors in gene therapy Cationic liposome Invitrogen (2012). "Lipofectamine 2000 Reagent...

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SV40

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modified SV40 based vectors as a viral vector for gene therapy. In these helper dependent virus or packaging cell line assisted produced vectors the SV40 large...

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Short hairpin RNA

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proved dangerous in past clinical trials. In the first generation of retro viral gene therapy, some patients treated with viral vectors for Wiskott–Aldrich...

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