Utility of In Vitro Mutagenesis of RPE65 Protein for Verification of Mutational Pathogenicity Before Gene Therapy.
Sign Up to like & getrecommendations! Published in 2019 at "JAMA ophthalmology"
DOI: 10.1001/jamaophthalmol.2019.3914
Abstract: Importance Next-generation sequencing can detect variants of uncertain significance (VUSs), for some of which gene therapy would not be advantageous. Therefore, the pathogenicity of compound heterozygous or homozygous variants should be confirmed before bilateral vitrectomy… read more here.
Keywords: vitro mutagenesis; gene therapy; rpe65; pathogenicity ... See more keywords
A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease: The Medicaid Perspective.
Sign Up to like & getrecommendations! Published in 2021 at "JAMA pediatrics"
DOI: 10.1001/jamapediatrics.2020.7140
Abstract: Importance Hundreds of gene therapies are undergoing clinical testing and are likely to be priced more than $1 million per course of treatment. The association that high prices will have with insurance coverage of gene… read more here.
Keywords: budget impact; gene; gene therapy; disease ... See more keywords
Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA.
Sign Up to like & getrecommendations! Published in 2023 at "Annals of clinical and translational neurology"
DOI: 10.1002/acn3.51772
Abstract: OBJECTIVE Mucopolysaccharidosis type IIIA (MPSIIIA) caused by recessive SGSH variants results in sulfamidase deficiency, leading to neurocognitive decline and death. No disease-modifying therapy is available. The AAVance gene therapy trial investigates AAVrh.10 overexpressing human sulfamidase… read more here.
Keywords: intracerebral gene; mucopolysaccharidosis; focal lesions; therapy ... See more keywords
Gene Therapy Based on Nucleic Acid Nanostructure.
Sign Up to like & getrecommendations! Published in 2020 at "Advanced healthcare materials"
DOI: 10.1002/adhm.202001046
Abstract: During the past decades, nucleic acids have been employed for the construction of versatile nanostructures with well-defined shapes and sizes. Owing to the remarkable programmability, addressability, and biocompatibility, nucleic acid nanostructures are extensively applied in… read more here.
Keywords: therapy based; gene; nucleic acid; gene therapy ... See more keywords
Red Blood Cell Anchoring Enables Targeted Transduction and Re‐Administration of AAV‐Mediated Gene Therapy
Sign Up to like & getrecommendations! Published in 2022 at "Advanced Science"
DOI: 10.1002/advs.202201293
Abstract: Adeno‐associated virus (AAV)‐mediated gene therapy is a promising therapeutic modality for curing many diseases including monogenic diseases. However, limited tissue‐targeting and restricted re‐administration due to the vector immunogenicity largely restrict its therapeutic potential. Here, using… read more here.
Keywords: aav mediated; administration; gene therapy; transduction ... See more keywords
Gene therapy decreases seizures in a model of Incontinentia pigmenti
Sign Up to like & getrecommendations! Published in 2017 at "Annals of Neurology"
DOI: 10.1002/ana.24981
Abstract: Incontinentia pigmenti (IP) is a genetic disease leading to severe neurological symptoms, such as epileptic seizures, but no specific treatment is available. IP is caused by pathogenic variants that inactivate the Nemo gene. Replacing Nemo… read more here.
Keywords: therapy decreases; gene therapy; incontinentia pigmenti; gene ... See more keywords
Gene therapy improves brain white matter in aromatic l‐amino acid decarboxylase deficiency
Sign Up to like & getrecommendations! Published in 2019 at "Annals of Neurology"
DOI: 10.1002/ana.25467
Abstract: Children with aromatic l‐amino acid decarboxylase (AADC) deficiency suffer from severe motor dysfunction. Restoration of dopamine levels in the putamen by gene therapy has led to significant improvement in motor function. This study explored brain… read more here.
Keywords: acid decarboxylase; aromatic amino; amino acid; gene therapy ... See more keywords
Optimizing VLP-production in Gene therapy: Opportunities and challenges for in silico modeling.
Sign Up to like & getrecommendations! Published in 2023 at "Biotechnology journal"
DOI: 10.1002/biot.202200636
Abstract: Over the past decades, virus-like particle (VLP)-based gene therapy (GT) evolved as a promising approach to cure inherited diseases or cancer. Tremendous costs due to inefficient production processes remain one of the key challenges despite… read more here.
Keywords: vlp production; production; gene therapy; cell lines ... See more keywords
Critical challenges and advances in recombinant adeno-associated virus (rAAV) biomanufacturing.
Sign Up to like & getrecommendations! Published in 2023 at "Biotechnology and bioengineering"
DOI: 10.1002/bit.28412
Abstract: Gene therapy is a promising therapeutic approach for genetic and acquired diseases nowadays. Among DNA delivery vectors, recombinant adeno-associated virus (rAAV) is one of the most effective and safest vectors used in commercial drugs and… read more here.
Keywords: raav; recombinant adeno; associated virus; adeno associated ... See more keywords
Gene therapy: A promising approach for breast cancer treatment
Sign Up to like & getrecommendations! Published in 2021 at "Cell Biochemistry and Function"
DOI: 10.1002/cbf.3676
Abstract: Breast cancer (BC) is the most prevalent malignancy and the second leading cause of death among women worldwide that is caused by numerous genetic and environmental factors. Hence, effective treatment for this type of cancer… read more here.
Keywords: cancer; breast cancer; gene therapy; gene ... See more keywords
The Potential Application of Branch‐PCR Assembled PTEN Gene Nanovector in Lung Cancer Gene Therapy
Sign Up to like & getrecommendations! Published in 2022 at "ChemBioChem"
DOI: 10.1002/cbic.202200387
Abstract: Gene therapy offers an alternative and promising avenue to lung cancer treatment. Here, we used dibenzocyclooctyne (DBCO)‐branched primers to construct a PTEN gene nanovector (NP‐PTEN) through branch‐PCR. NP‐PTEN showed the nanoscale structure with biocompatible size… read more here.
Keywords: lung cancer; pten gene; gene therapy; gene nanovector ... See more keywords