Integrated CMC Development of Gene Therapies

In this webinar, learn strategies to accelerate analytical and formulation development of gene therapy drugs, characterization approaches for drugs containing both protein and nucleic acids, and...
Source: The Scientist - Category: Science Tags: The Marketplace The Scientist Source Type: news

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Two papers in this issue highlight gene therapy for  skin fragility diseases. March and colleagues employed the transcription activator-like effector nuclease to disrupt dominant-negative epidermolytic ichthyosis–causing mutations in keratin 10 (KRT10) alleles in keratinocytes, showing abrogation of disease phenotypes in a murine model. Takashima and colleagues employed a different nuclease system, CRISPR/Cas9, to co-opt non-homologous end-joining to correct a common frameshift mutation in one of the alleles of the collagen type VII (COL7A1) gene, restoring COL7A1 expression in recessive dystrophic epidermolysis...
Source: Journal of Investigative Dermatology - Category: Dermatology Tags: Editorial Source Type: research
Ex  vivo gene therapy is a promising approach to treat devastating skin fragility diseases. March et al. and Takashima et al. report that programmable nucleases—TALENs and CRISPR/Cas9—can safely and efficiently correct genetic defects in cultured adult skin cells, paving the way for broader clin ical applications of gene therapies in dermatology.
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Commentary Source Type: research
In conclusion, PEI-CeO2 nanocarriers could act as a potential candidate for gene and drug delivery to cancerous and tumor cells.Graphical abstract
Source: Life Sciences - Category: Biology Source Type: research
Abstract Adeno-associated virus (AAV) vectors are an efficient method of gene delivery to various tissues including the lung. Mouse models are often used as a preliminary preclinical model in order to advance AAV lung gene therapy vectors. In this chapter we describe an AAV purification protocol using heparin affinity chromatography as well as an intranasal and intratracheal method of delivering AAV vectors to the lungs of mice. PMID: 30783985 [PubMed - indexed for MEDLINE]
Source: Mol Biol Cell - Category: Molecular Biology Authors: Tags: Methods Mol Biol Source Type: research
Abstract Adeno-associated virus (AAV) has emerged as the vector of choice for delivering genes to the retina. Indeed, the first gene therapy to receive FDA approval in the United States is an AAV-based treatment for the inherited retinal disease, Leber congenital amaurosis-2. Voretigene neparvovec (Luxturna™) is delivered to patients via subretinal (SR) injection, an invasive surgical procedure that requires detachment of photoreceptors (PRs) from the retinal pigment epithelium (RPE). It has been reported that subretinal administration of vector under the cone-exclusive fovea leads to a loss of central retin...
Source: Mol Biol Cell - Category: Molecular Biology Authors: Tags: Methods Mol Biol Source Type: research
Abstract Techniques to localize vector transgenes in cells and tissues are essential in order to fully characterize gene therapy outcomes. In situ hybridization (ISH) uses synthesized complementary RNA or DNA nucleotide probes to localize and detect sequences of interest in fixed cells, tissue sections, or whole tissue mounts. Variations in techniques include adding labels to probes, such as fluorophores, which can allow for the simultaneous visualization of multiple targets. Here we provide the steps necessary to: (1) label probes for colorimetric visualization and (2) perform ISH on OCT cryo-preserved fixed froz...
Source: Mol Biol Cell - Category: Molecular Biology Authors: Tags: Methods Mol Biol Source Type: research
Abstract The adeno-associated viral vector (AAV) platform has developed into a primary modality for efficient in vivo, and in more limited settings, in vitro or ex vivo gene transfer. Its applications range from a tool for experimental purposes to preclinical and clinical gene therapy. The ability to accurately and reproducibly quantify vector concentration is critical for any of these applications. While several quantification assays are available, here we outline a detailed protocol for the quantification of DNase-I protected vector genomes reliant on the polymerase chain reaction (PCR) as a measure of the activ...
Source: Mol Biol Cell - Category: Molecular Biology Authors: Tags: Methods Mol Biol Source Type: research
Cancer Gene Therapy, Published online: 18 July 2019; doi:10.1038/s41417-019-0124-8Viral vector: potential therapeutic for glioblastoma multiforme
Source: Cancer Gene Therapy - Category: Cancer & Oncology Authors: Source Type: research
Cancer Gene Therapy, Published online: 18 July 2019; doi:10.1038/s41417-019-0123-9The interaction between microRNA-152 and DNA methyltransferase-1 as an epigenetic prognostic biomarker in HCV-induced liver cirrhosis and HCC patients
Source: Cancer Gene Therapy - Category: Cancer & Oncology Authors: Source Type: research
A promising HIV vaccine developed at Oregon Health&Science University is getting closer to human clinical trials. Dr. Louis Picker, a professor at OHSU’s Vaccine&Gene Therapy Institute, said his lab has tested a weakened version of the vaccine in monkeys that provides similar protection to the original, stronger version. Developing a weaker, or “live-attenuated,” version is important to ensure safety for people with weakened immune systems, Picker said. The new version eliminated SIV,…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
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