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Therapy: Gene Therapy

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Total 5568 results found since Jan 2013.

Cold atmospheric plasma mediated cell membrane permeation and gene delivery-empirical interventions and pertinence
Adv Colloid Interface Sci. 2023 Aug 28;320:102989. doi: 10.1016/j.cis.2023.102989. Online ahead of print.ABSTRACTDelivery of genetic material to cells is an integral tool to analyze and reveal the genetic interventions in normal cellular processes and differentiation, disease development and for gene therapy. It has profound applications in pharmaceutical, agricultural, environmental and biotechnological sectors. The major methods relied for gene delivery or transfection requires either viral vectors or xenogenic carrier molecules, which renders probabilistic carcinogenic, immunogenic and toxic effects. A newly evolved phy...
Source: Advances in Colloid and Interface Science - September 7, 2023 Category: Chemistry Authors: P R Sreedevi K Suresh Source Type: research

Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in the Phase 3 Trial GENEr8-1
CONCLUSION: Valoctocogene roxaparvovec provided clinically meaningful HRQOL improvement for men with severe HA.PMID:37678546 | DOI:10.1016/j.jtha.2023.08.032
Source: Thrombosis and Haemostasis - September 7, 2023 Category: Hematology Authors: Brian O'Mahony Amy L Dunn Andrew D Leavitt Flora Peyvandi Margareth C Ozelo Johnny Mahlangu Kathelijne Peerlinck Jiaan-Der Wang Gillian C Lowe Chee Wee Tan Adam Giermasz Huyen Tran Teh-Liane Khoo Erin Cockrell Dominic Pepperell Herv é Chambost Maria Fern Source Type: research

Engineered adult stem cells: Current clinical trials status of disease treatment
Prog Mol Biol Transl Sci. 2023;199:33-62. doi: 10.1016/bs.pmbts.2023.02.007. Epub 2023 Jul 26.ABSTRACTRegenerative medicine is an interdisciplinary field involving the process of replacing and regenerating cells/tissues or organs by integrating medicine, science, and engineering principles to enhance the intrinsic regenerative capacity of the host. Recently, engineered adult stem cells have gained attention for their potential use in regenerative medicine by reducing inflammation and modulating the immune system. This chapter introduces adult stem cell engineering and chimeric antigen receptor T cells (CAR T) gene therapy ...
Source: Mol Biol Cell - September 7, 2023 Category: Molecular Biology Authors: Pingping Han Corey Stephan Moran Chun Liu Rebecca Griffiths Yinghong Zhou Sa šo Ivanovski Source Type: research

Stimuli-Responsive Hydrogel Microcapsules Harnessing the COVID-19 Immune Response for Cancer Therapeutics
We present the development of stimuli-responsive microcapsules, loaded with a viral immunogenetic agent, harnessing the immune response against the Coronavirus Disease 2019, COVID-19, to selectively attack liver cancer cells (hepatoma) or recognize breast cancer or hepatoma, by expression of green fluorescence protein, GFP. The pH-responsive microcapsules, modified with DNA-tetrahedra nanostructures, increased hepatoma permeation by 50%. Incorporation of a GFP-encoding lentivirus vector inside the tumor-targeting pH-stimulated miRNA-triggered and Alpha-fetoprotein-dictated microcapsules enables the demonstration of neoplas...
Source: Angewandte Chemie - September 7, 2023 Category: Chemistry Authors: Amit Fischer Avner Ehrlich Yevgeni Plotkin Yu Ouyang Klil Asulin Ioannidis Konstantinos Chunhai Fan Yaakov Nahmias Itamar Willner Source Type: research

Cold atmospheric plasma mediated cell membrane permeation and gene delivery-empirical interventions and pertinence
Adv Colloid Interface Sci. 2023 Aug 28;320:102989. doi: 10.1016/j.cis.2023.102989. Online ahead of print.ABSTRACTDelivery of genetic material to cells is an integral tool to analyze and reveal the genetic interventions in normal cellular processes and differentiation, disease development and for gene therapy. It has profound applications in pharmaceutical, agricultural, environmental and biotechnological sectors. The major methods relied for gene delivery or transfection requires either viral vectors or xenogenic carrier molecules, which renders probabilistic carcinogenic, immunogenic and toxic effects. A newly evolved phy...
Source: Advances in Colloid and Interface Science - September 7, 2023 Category: Chemistry Authors: P R Sreedevi K Suresh Source Type: research

Engineered adult stem cells: Current clinical trials status of disease treatment
Prog Mol Biol Transl Sci. 2023;199:33-62. doi: 10.1016/bs.pmbts.2023.02.007. Epub 2023 Jul 26.ABSTRACTRegenerative medicine is an interdisciplinary field involving the process of replacing and regenerating cells/tissues or organs by integrating medicine, science, and engineering principles to enhance the intrinsic regenerative capacity of the host. Recently, engineered adult stem cells have gained attention for their potential use in regenerative medicine by reducing inflammation and modulating the immune system. This chapter introduces adult stem cell engineering and chimeric antigen receptor T cells (CAR T) gene therapy ...
Source: Mol Biol Cell - September 7, 2023 Category: Molecular Biology Authors: Pingping Han Corey Stephan Moran Chun Liu Rebecca Griffiths Yinghong Zhou Sa šo Ivanovski Source Type: research

Cold atmospheric plasma mediated cell membrane permeation and gene delivery-empirical interventions and pertinence
Adv Colloid Interface Sci. 2023 Aug 28;320:102989. doi: 10.1016/j.cis.2023.102989. Online ahead of print.ABSTRACTDelivery of genetic material to cells is an integral tool to analyze and reveal the genetic interventions in normal cellular processes and differentiation, disease development and for gene therapy. It has profound applications in pharmaceutical, agricultural, environmental and biotechnological sectors. The major methods relied for gene delivery or transfection requires either viral vectors or xenogenic carrier molecules, which renders probabilistic carcinogenic, immunogenic and toxic effects. A newly evolved phy...
Source: Advances in Colloid and Interface Science - September 7, 2023 Category: Chemistry Authors: P R Sreedevi K Suresh Source Type: research

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