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Therapy: Gene Therapy

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Total 490 results found since Jan 2013.

Advances and Challenges of Stimuli-Responsive Nucleic Acids Delivery System in Gene Therapy
Pharmaceutics. 2023 May 10;15(5):1450. doi: 10.3390/pharmaceutics15051450.ABSTRACTGene therapy has emerged as a powerful tool to treat various diseases, such as cardiovascular diseases, neurological diseases, ocular diseases and cancer diseases. In 2018, the FDA approved Patisiran (the siRNA therapeutic) for treating amyloidosis. Compared with traditional drugs, gene therapy can directly correct the disease-related genes at the genetic level, which guarantees a sustained effect. However, nucleic acids are unstable in circulation and have short half-lives. They cannot pass through biological membranes due to their high mole...
Source: Cancer Control - May 27, 2023 Category: Cancer & Oncology Authors: Meng Lin Xianrong Qi Source Type: research

Biomedical applications of nanomaterials in the advancement of nucleic acid therapy: Mechanistic challenges, delivery strategies, and therapeutic applications
Int J Biol Macromol. 2023 Apr 26:124582. doi: 10.1016/j.ijbiomac.2023.124582. Online ahead of print.ABSTRACTIn the past few decades, substantial advancement has been made in nucleic acid (NA)-based therapies. Promising treatments include mRNA, siRNA, miRNA, and anti-sense DNA for treating various clinical disorders by modifying the expression of DNA or RNA. However, their effectiveness is limited due to their concentrated negative charge, instability, large size, and host barriers, which make widespread application difficult. The effective delivery of these medicines requires safe vectors that are efficient & selective...
Source: International Journal of Biological Macromolecules - April 28, 2023 Category: Biochemistry Authors: Krishna Yadav Kantrol Kumar Sahu None Sucheta S Princely Ebenezer Gnanakani Pavani Sure R Vijayalakshmi V D Sundar Versha Sharma Ruchita Antil Megha Jha Sunita Minz Anindya Bagchi Madhulika Pradhan Source Type: research

Suppression of mTOR expression by siRNA leads to cell cycle arrest and apoptosis induction in MDA-MB-231 breast cancer cells
CONCLUSION: With these results, we can conclude that mTOR-siRNA exerts direct 'anti-breast cancer' activity propagated by the S6K-GSK-3β- caspase 3 mediated apoptosis and by inducing cell cycle arrest.PMID:36999418 | DOI:10.2174/1566523223666230329085606
Source: Current Gene Therapy - March 31, 2023 Category: Genetics & Stem Cells Authors: Roja Sahu Shivesh Jha Shakti Pattanayak Source Type: research

A cathepsin B/GSH dual-responsive fluorinated peptide for effective siRNA delivery to cancer cells
Bioorg Chem. 2023 Mar 20;135:106485. doi: 10.1016/j.bioorg.2023.106485. Online ahead of print.ABSTRACTSmall interfering RNA (siRNA) can be exploited to silence specific genes associated with cancer development, and successful siRNA therapy is highly dependent on the efficiency of the siRNA delivery vector. Herein, a well-designed novel redox- and enzyme-responsive fluorinated polyarginine (PFC-PR) was developed to be used as an anti-cancer siRNA carrier. The multiple guanidine groups could provide positive charges and bind with siRNA efficiently, and further fluorination modification enhanced the interaction with siRNA, re...
Source: Bioorganic Chemistry - March 24, 2023 Category: Chemistry Authors: Zhen Shi Yuhan Yang Ziyang Guo Shun Feng Yu Wan Source Type: research

Carbon Dots with Guanidinium and Amino Acid Functional Groups for Targeted Small Interfering RNA Delivery toward Tumor Gene Therapy
Large amino acid mimicking carbon dots with guanidinium functionalization (LAAM GUA-CDs) can be prepared to carry small interfering RNA by multiple hydrogen bonds and target to tumor tissues specifically through LAT1 receptor. After loading siBcl-2 as a therapeutic agent, LAAM GUA-CDs/siBcl-2 bring about superior antitumor effects without side effects during the gene therapy. AbstractSmall interfering RNA (siRNA)-based gene therapy represents a promising strategy for tumor treatment. Novel gene vectors that can achieve targeted delivery of siRNA to the tumor cells without causing any side effects are urgently needed. To th...
Source: Small - February 25, 2023 Category: Nanotechnology Authors: Huimin Xu, Jianqiao Chang, Hao Wu, Haoyu Wang, Wenjing Xie, Yunchao Li, Xiaohong Li, Yang Zhang, Louzhen Fan Tags: Research Article Source Type: research

Engineered NF- κB siRNA-encapsulating exosomes as a modality for therapy of skin lesions
DiscussionOverall, this study establishes a feasible therapeutic strategy for skin injury, which may offer an alternative to conventional biological therapies requiring two or more independent compounds.
Source: Frontiers in Immunology - February 8, 2023 Category: Allergy & Immunology Source Type: research

Synergistic treatment of osteosarcoma with biomimetic nanoparticles transporting doxorubicin and siRNA
ConclusionDOX/siSUR-PLGA@MSCM NPs can show improved therapeutic effects in osteosarcoma patients due to the combination of a chemotherapeutic drug and gene therapy based on their good tumor targeting and biosafety.
Source: Frontiers in Oncology - January 23, 2023 Category: Cancer & Oncology Source Type: research