Notes in j. Gene Therapy

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Published	07/28/2024	Gene therapy is the {{c1::modification of the endogenous DNA and/or RNA in cells}}, most often based on the {{c2::introduction of exogenous nucle…
Published	07/28/2024	Gene therapy is divided into three categories {{c1:: gene addition or augmentation (increase levels of an mRNA and/or protein)}}, {{c2:: Gene expressi…
Published	07/28/2024	Gene therapy can occur {{c1::in vivo}} or {{c1::ex vivo}}
Published	07/28/2024	Gene therapy requirements include
Published	07/28/2024	Nucleic acid delivery approaches include {{c1::viral vector-based approaches}} and {{c1::non-viral vector-based approaches}}
Published	07/28/2024	Explain the process of converting a virus into a vector
Published	07/28/2024	Barriers of viral vector based gene therapy include
Published	07/28/2024	Most commonly used viral vectors are {{c1::retrovirus/lentivirus}} and {{c1::adeno-associated virus}} rather than the Adenovirus previously used
Published	07/28/2024	Mutations in the Hemophelia B gene which lead to deficiency in Factor IX in the past were only effectively treated by {{c1::recombinant Factor IX (inj…
Published	07/28/2024	Leber's congenital amaurosis which causes {{c1::blindness}} with onset during {{c1::childhood}} due to mutation in {{c2::RPE65 gene}} was shown to be …
Published	07/28/2024	Treating severe combined immunodeficiency (SCID) is an {{c1::ex-vivo}} example of hematopoietic stem cell gene therapy where an {{c1::ex-vivo cul…
Published	07/28/2024	Treatment of Duchenne Muscle Dystrophy via gene therapy is an introduction of an {{c1::anti sense oligonucleotide}} into DNA that would block the {{c2…
Published	07/28/2024	Clinical trials for treating DMD with {{c1::oligonucleotides that promote skipping of exon 51}} showed promise but ultimately led to absence in clinic…
Published	07/28/2024	Spinal muscular atrophy is an {{c1::autosomal recessive::inheritance pattern}} disease and very common cause of death in newborn children due to …
Published	07/28/2024	Zolgensma is a more advanced treatment for spinal muscular atrophy involving a {{c1::by an IV injection of an AAV therapy that increases expression of…
Published	07/28/2024	The treatment of Alpha-1 Anti-trypsin (A1AT) deficiency which causes 10% of patients to develop liver disease with progression to cirrhosis is an exam…
Published	07/28/2024	CRISPR-Cas9 is an approach where you can {{c1::cut out mutations}} in DNA sequence and {{c2::paste corrected sequence}} by homology directed repair to…
Published	07/28/2024	Most cutting edge treatment of DMD involves using {{c1::CRISPR-Cas9 in an AAV}} to splice out {{c1::all common mutations that lead to premature stop c…
Published	07/28/2024	DMD can be treated by using {{c1::CRISPR-Cas9 in an AAV to splice out all common mutations that lead to premature stop codons}}
Published	07/28/2024	Transduction of exogenously genetically engineered T Cells that express a {{c1::chimaeric antigen receptor}} that recognizes a specific cancer antigen…
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