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Therapy: Gene Therapy
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Total 5568 results found since Jan 2013.
CRISPR applications in cancer diagnosis and treatment
Cell Mol Biol Lett. 2023 Sep 6;28(1):73. doi: 10.1186/s11658-023-00483-4.ABSTRACTCancer remains a significant global health challenge, necessitating the exploration of novel and more precise therapeutic options beyond conventional treatments. In this regard, clustered regularly interspaced short palindromic repeats (CRISPR) systems have emerged as highly promising tools for clinical gene editing applications. The CRISPR family encompasses diverse CRISPR-associated (Cas) proteins that possess the ability to recognize specific target sequences. The initial CRISPR system consisted of the Cas9 protein and a single-guide RNA, w...
Source: Cellular and Molecular Biology Letters - September 6, 2023 Category: Biochemistry Authors: Mingxia Wang Menghui Chen Xia Wu Xinbo Huang Bo Yu Source Type: research
Multi-attribute analysis of adeno-associated virus by size exclusion chromatography with fluorescence and triple-wavelength UV detection
Anal Biochem. 2023 Sep 2:115311. doi: 10.1016/j.ab.2023.115311. Online ahead of print.ABSTRACTAdeno-associated virus (AAV) is the leading platform for in vivo gene therapy to treat numerous genetic diseases. Comprehensive analysis of the AAV particles is essential to ensure desired safety and efficacy. An array of techniques is required to evaluate their critical quality attributes. However, many of these techniques are expensive, time-consuming, labour-intensive, and varying in accuracy. Size exclusion chromatography coupled with fluorescence and triple-wavelength ultraviolet detection (SEC-FLD-TWUV) and incorporating an ...
Source: Analytical Biochemistry - September 4, 2023 Category: Biochemistry Authors: Yongjing Xie Michael Butler Source Type: research
Proof of concept of intracochlear drug administration by laser-assisted bioprinting in mice
Hear Res. 2023 Aug 29;438:108880. doi: 10.1016/j.heares.2023.108880. Online ahead of print.ABSTRACTTranstympanic administration is used clinically for the injection of gentamicin and/or corticosteroids. This atraumatic route is based on passive diffusion through the round window membrane (RWM). The main limitation of this method is related to the clearance through the Eustachian tube, making the concentration of the therapeutic agent at the intracochlear level uncertain and limited. Moreover, this technique remains unsuitable for molecules of high molecular weight or in the case of gene therapies. The purpose was to study ...
Source: Hearing Research - September 4, 2023 Category: Audiology Authors: Manon Jaffredo Oc éane Duchamp Nicolas Touya Yohan Bouleau Didier Dulon Raphael Devillard Damien Bonnard Source Type: research
Therapeutic potential of gene therapy for gastrointestinal diseases: Advancements and future perspectives
Mol Ther Oncolytics. 2023 Aug 18;30:193-215. doi: 10.1016/j.omto.2023.08.007. eCollection 2023 Sep 21.ABSTRACTAdvancements in understanding the pathogenesis mechanisms underlying gastrointestinal diseases, encompassing inflammatory bowel disease, gastrointestinal cancer, and gastroesophageal reflux disease, have led to the identification of numerous novel therapeutic targets. These discoveries have opened up exciting possibilities for developing gene therapy strategies to treat gastrointestinal diseases. These strategies include gene replacement, gene enhancement, gene overexpression, gene function blocking, and transgenic...
Source: Cancer Control - September 4, 2023 Category: Cancer & Oncology Authors: Ning-Ning Yue Hao-Ming Xu Jing Xu Min-Zheng Zhu Yuan Zhang Cheng-Mei Tian Yu-Qiang Nie Jun Yao Yu-Jie Liang De-Feng Li Li-Sheng Wang Source Type: research
Multi-attribute analysis of adeno-associated virus by size exclusion chromatography with fluorescence and triple-wavelength UV detection
Anal Biochem. 2023 Sep 2:115311. doi: 10.1016/j.ab.2023.115311. Online ahead of print.ABSTRACTAdeno-associated virus (AAV) is the leading platform for in vivo gene therapy to treat numerous genetic diseases. Comprehensive analysis of the AAV particles is essential to ensure desired safety and efficacy. An array of techniques is required to evaluate their critical quality attributes. However, many of these techniques are expensive, time-consuming, labour-intensive, and varying in accuracy. Size exclusion chromatography coupled with fluorescence and triple-wavelength ultraviolet detection (SEC-FLD-TWUV) and incorporating an ...
Source: Analytical Biochemistry - September 4, 2023 Category: Biochemistry Authors: Yongjing Xie Michael Butler Source Type: research
Proof of concept of intracochlear drug administration by laser-assisted bioprinting in mice
Hear Res. 2023 Aug 29;438:108880. doi: 10.1016/j.heares.2023.108880. Online ahead of print.ABSTRACTTranstympanic administration is used clinically for the injection of gentamicin and/or corticosteroids. This atraumatic route is based on passive diffusion through the round window membrane (RWM). The main limitation of this method is related to the clearance through the Eustachian tube, making the concentration of the therapeutic agent at the intracochlear level uncertain and limited. Moreover, this technique remains unsuitable for molecules of high molecular weight or in the case of gene therapies. The purpose was to study ...
Source: Hearing Research - September 4, 2023 Category: Audiology Authors: Manon Jaffredo Oc éane Duchamp Nicolas Touya Yohan Bouleau Didier Dulon Raphael Devillard Damien Bonnard Source Type: research
Multi-attribute analysis of adeno-associated virus by size exclusion chromatography with fluorescence and triple-wavelength UV detection
Anal Biochem. 2023 Sep 2:115311. doi: 10.1016/j.ab.2023.115311. Online ahead of print.ABSTRACTAdeno-associated virus (AAV) is the leading platform for in vivo gene therapy to treat numerous genetic diseases. Comprehensive analysis of the AAV particles is essential to ensure desired safety and efficacy. An array of techniques is required to evaluate their critical quality attributes. However, many of these techniques are expensive, time-consuming, labour-intensive, and varying in accuracy. Size exclusion chromatography coupled with fluorescence and triple-wavelength ultraviolet detection (SEC-FLD-TWUV) and incorporating an ...
Source: Analytical Biochemistry - September 4, 2023 Category: Biochemistry Authors: Yongjing Xie Michael Butler Source Type: research
In Vivo CRISPR Gene Editing in Patients with Herpes Stromal Keratitis
Mol Ther. 2023 Aug 31:S1525-0016(23)00487-2. doi: 10.1016/j.ymthe.2023.08.021. Online ahead of print.ABSTRACTIn vivo CRISPR gene therapy holds large clinical potential, but the safety and efficacy remain largely unknown. Here, we injected a single dose of HSV-1-targeting CRISPR formulation in the cornea of three patients with severe refractory herpes stromal keratitis (HSK) during corneal transplantation. Our study is an investigated initiated, open-label, single-arm, non-randomized interventional trial at a single center (NCT04560790). We found neither detectable CRISPR-induced off-target cleavages by GUIDE-seq nor system...
Source: Herpes - September 2, 2023 Category: Infectious Diseases Authors: Anji Wei Di Yin Zimeng Zhai Sikai Ling Huangying Le Lijia Tian Jianjiang Xu Soren R Paludan Yujia Cai Jiaxu Hong Source Type: research
Combined cytotoxic and immune-stimulatory gene therapy for primary adult high-grade glioma: a phase 1, first-in-human trial
This study aimed to evaluate the safety and activity of two adenoviral vectors expressing HSV1-TK (Ad-hCMV-TK) and Flt3L (Ad-hCMV-Flt3L) in patients with high-grade glioma.METHODS: In this dose-finding, first-in-human trial, treatment-naive adults aged 18-75 years with newly identified high-grade glioma that was evaluated per immunotherapy response assessment in neuro-oncology criteria, and a Karnofsky Performance Status score of 70 or more, underwent maximal safe resection followed by injections of adenoviral vectors expressing HSV1-TK and Flt3L into the tumour bed. The study was conducted at the University of Michigan Me...
Source: Ann Oncol - September 1, 2023 Category: Cancer & Oncology Authors: Yoshie Umemura Daniel Orringer Larry Junck Maria L Varela Molly E J West Syed M Faisal Andrea Comba Jason Heth Oren Sagher Denise Leung Aaron Mammoser Shawn Hervey-Jumper Daniel Zamler Viveka N Yadav Patrick Dunn Wajd Al-Holou Todd Hollon Michelle M Kim D Source Type: research
GCH1: GTP cyclohydroxylase1: Role in neurodegenerative diseases
Gene. 2023 Aug 29:147749. doi: 10.1016/j.gene.2023.147749. Online ahead of print.ABSTRACTGCH1 gene provides directions for the synthesis of GTP cyclohydrolase 1 which regulates the formation of Tetrahydrobiopterin (BH4). BH4 is a crucial cofactor for essential neurotransmitters synthesis such as dopamine, serotonin and nitric oxide synthases. Deficiency of GCH1 limits the synthesis of BH4 which is responsible for neuropsychiatric diseases such as dopa-responsive dystonia, hyperalaninemia, Parkinson's disease and depression. Few single nucleotide polymorphisms of GCH1 gene are also responsible for pain in Sickle cell diseas...
Source: Gene - August 31, 2023 Category: Genetics & Stem Cells Authors: Parul Gupta Ravindra Kumar Source Type: research
A shift in focus towards precision oncology, driven by revolutionary nanodiagnostics; revealing mysterious pathways in colorectal carcinogenesis
This article examines current nanomedicine patents for CRC, including the work of Delta-Fly, Merrimack, and Pfenning, Meaning & Partner, among others. In terms of future nanomedicine research and development, ligand production, particle size, and clearance are crucial factors. Lastly, the numerous nanostructures utilized in nanomedicine for targeted drug administration and diagnostics indicate optimistic prospects for enhancing CRC treatment. The successes of nanomedicine research and development for existing colon cancer treatments are also highlighted in this review.PMID:37650995 | DOI:10.1007/s00432-023-05331-8
Source: Clinical Colorectal Cancer - August 31, 2023 Category: Cancer & Oncology Authors: Satyam Sharma Sankha Bhattacharya Kajal Joshi Sanjiv Singh Source Type: research
