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Therapy: Gene Therapy
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Total 490 results found since Jan 2013.
A comprehensive update of siRNA delivery design strategies for targeted and effective gene silencing in gene therapy and other applications
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Source: Expert Opinion on Drug Discovery - December 17, 2022 Category: Drugs & Pharmacology Authors: Ahmed Khaled Abosalha Waqar Ahmad Jacqueline Boyajian Paromita Islam Merry Ghebretatios Sabrina Schaly Rahul Thareja Karan Arora Satya Prakash Source Type: research
siAKR1C3@PPA complex nucleic acid nanoparticles inhibit castration-resistant prostate cancer in vitro
ConclusionWe concluded that siAKR1C3@PPA may arrest the cell cycle and affect cell proliferation.
Source: Frontiers in Oncology - December 16, 2022 Category: Cancer & Oncology Source Type: research
A hybrid nanoassembly for ultrasound-inducible cytosolic siRNA delivery and cancer sono-gene therapy
Ultrason Sonochem. 2022 Dec 9;92:106262. doi: 10.1016/j.ultsonch.2022.106262. Online ahead of print.NO ABSTRACTPMID:36512940 | DOI:10.1016/j.ultsonch.2022.106262
Source: Ultrasonics Sonochemistry - December 13, 2022 Category: Chemistry Authors: Gaigai Li Yan Zhang Jinbo Li Source Type: research
CNS Delivery of Nucleic Acid Therapeutics: Beyond the Blood –Brain Barrier and Towards Specific Cellular Targeting
AbstractNucleic acid-based therapeutic molecules including small interfering RNA (siRNA), microRNA(miRNA), antisense oligonucleotides (ASOs), messenger RNA (mRNA), and DNA-based gene therapy have tremendous potential for treating diseases in the central nervous system (CNS). However, achieving clinically meaningful delivery to the brain and particularly to target cells and sub-cellular compartments is typically very challenging. Mediating cell-specific delivery in the CNS would be a crucial advance that mitigates off-target effects and toxicities. In this review, we describe these challenges and provide contemporary eviden...
Source: Pharmaceutical Research - November 15, 2022 Category: Drugs & Pharmacology Source Type: research
Peptidyl Virus ‐Like Nanovesicles as Reconfigurable “Trojan Horse” for Targeted siRNA Delivery and Synergistic Inhibition of Cancer Cells
Peptidyl virus-like nanovesicles (pVLNs) composed of bilayer membranes are assembled by the peptides and siRNA. The targeting and enzyme-responsive sequences on the bilayer's surface allow the pVLNs to enter cancer cells with high efficiency and control the release of genetic drugs in response to the subcellular environment. The resulting gene silencing efficiency is 92% and cancer cell mortality is 96%. AbstractThe self-assembly of peptidyl virus-like nanovesicles (pVLNs) composed of highly ordered peptide bilayer membranes that encapsulate the small interfering RNA (siRNA) is reported. The targeting and enzyme-responsive...
Source: Small - November 14, 2022 Category: Nanotechnology Authors: Zixuan Wang,
Dongzhao Hao,
Yuefei Wang,
Jinwu Zhao,
Jiaxing Zhang,
Xi Rong,
Jiaojiao Zhang,
Jiwei Min,
Wei Qi,
Rongxin Su,
Mingxia He Tags: Research Article Source Type: research
Peptidyl Virus-Like Nanovesicles as Reconfigurable "Trojan Horse" for Targeted siRNA Delivery and Synergistic Inhibition of Cancer Cells
Small. 2022 Nov 13:e2204959. doi: 10.1002/smll.202204959. Online ahead of print.ABSTRACTThe self-assembly of peptidyl virus-like nanovesicles (pVLNs) composed of highly ordered peptide bilayer membranes that encapsulate the small interfering RNA (siRNA) is reported. The targeting and enzyme-responsive sequences on the bilayer's surface allow the pVLNs to enter cancer cells with high efficiency and control the release of genetic drugs in response to the subcellular environment. By transforming its structure in response to the highly expressed enzyme matrix metalloproteinase 7 (MMP-7) in cancer cells, it helps the siRNA esca...
Source: Cancer Control - November 13, 2022 Category: Cancer & Oncology Authors: Zixuan Wang Dongzhao Hao Yuefei Wang Jinwu Zhao Jiaxing Zhang Xi Rong Jiaojiao Zhang Jiwei Min Wei Qi Rongxin Su Mingxia He Source Type: research
Mechanisms and challenges of nanocarriers as non-viral vectors of therapeutic genes for enhanced pulmonary delivery
J Control Release. 2022 Nov 10:S0168-3659(22)00739-8. doi: 10.1016/j.jconrel.2022.10.061. Online ahead of print.ABSTRACTWith the rapid development of biopharmaceuticals and the outbreak of COVID-19, the world has ushered in a frenzy to develop gene therapy. Therefore, therapeutic genes have received enormous attention. However, due to the extreme instability and low intracellular gene expression of naked genes, specific vectors are required. Viral vectors are widely used attributed to their high transfection efficiency. However, due to the safety concerns of viral vectors, nanotechnology-based non-viral vectors have attrac...
Source: Cancer Control - November 13, 2022 Category: Cancer & Oncology Authors: Hezhi Wang Lu Qin Xin Zhang Jian Guan Shirui Mao Source Type: research
Current advancements in self-assembling nanocarriers-based siRNA delivery for cancer therapy
Colloids Surf B Biointerfaces. 2022 Nov 2;221:113002. doi: 10.1016/j.colsurfb.2022.113002. Online ahead of print.ABSTRACTDifferent therapeutic practices for treating cancers have significantly evolved to compensate and/or overcome the failures in conventional methodologies. The demonstrated potentiality in completely inhibiting the tumors and in preventing cancer relapse has made nucleic acids therapy (NAT)/gene therapy as an attractive practice. This has been made possible because NAT-based cancer treatments are highly focused on the fundamental mechanisms - i.e., silencing the expression of oncogenic genes responsible fo...
Source: Colloids and Surfaces - November 12, 2022 Category: Biotechnology Authors: Ganeshlenin Kandasamy Dipak Maity Source Type: research
Recent advances in gene therapy: genetic bullets to the root of the problem
AbstractGenetics and molecular genetic techniques have changed many perspectives and paradigms in medicine. Using genetic methods, many diseases have been cured or alleviated. Gene therapy, in its simplest definition, is application of genetic materials and related techniques to treat various human diseases. Evaluation of the trends in the field of medicine and therapeutics clarifies that gene therapy has attracted a lot of attention due to its powerful potential to treat a number of diseases. There are various genetic materials that can be used in gene therapy such as DNA, single- and double-stranded RNA, siRNA and shRNA....
Source: Clinical and Experimental Medicine - October 25, 2022 Category: Research Source Type: research
Long non-coding RNA LUCAT1 regulates the RAS pathway to promote the proliferation and invasion of malignant glioma cells through ABCB1
In this study, the role of lung cancer associated transcript 1 (lncRNA LUCAT 1) in glioma occurrence and development, as well as its possible regulatory mechanism, was explored. We utilized the gene chip technology in the preliminary experiment, and based on the experiment results, selected LUCAT1（NONHSAT102745）, which was significantly upregulated in glioma, and ATP-binding cassette Subfamily B member l (ABCB1), which was significantly down-regulated in co-expression analysis, for study. Next, the expression of LUCAT1 and ABCB1 in cells and tissues was immediately evaluated. Subsequently, the cells were transfected wi...
Source: Cell Research - October 21, 2022 Category: Cytology Authors: Xia Wu Lvmeng Song Xiangrong Chen Yalan Zhang Shun Li Xiaoping Tang Source Type: research
RNA interference (RNAi)-based therapeutics for treatment of rare neurologic diseases
Mol Aspects Med. 2022 Oct 15:101148. doi: 10.1016/j.mam.2022.101148. Online ahead of print.ABSTRACTAdvances in genome sequencing have greatly facilitated the identification of genomic variants underlying rare neurodevelopmental and neurodegenerative disorders. Understanding the fundamental causes of rare monogenic disorders has made gene therapy a possible treatment approach for these conditions. RNA interference (RNAi) technologies such as small interfering RNA (siRNA), microRNA (miRNA), and short hairpin RNA (shRNA), and other oligonucleotide-based modalities such as antisense oligonucleotides (ASOs) are being developed ...
Source: Molecular Medicine - October 18, 2022 Category: Molecular Biology Authors: Noelle D Germain Wendy K Chung Patrick D Sarmiere Source Type: research
