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Source: Mol Biol Cell
Therapy: Gene Therapy
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Total 71 results found since Jan 2013.
Integrated bioinformatics analysis of microarray data from non-small cell lung cancer
In conclusion, the identification of hsa-miR-21, hsa-miR-141, hsa-miR-200b hsa-miR-30a, hsa-miR-30d and hsa-miR-145 provides a new theoretical basis for understanding the development of NSCLC.PMID:37715441 | DOI:10.14715/cmb/2023.69.7.35
Source: Mol Biol Cell - September 16, 2023 Category: Molecular Biology Authors: Lulu Feng Wenping Cai Shan Jin Caipu Chun Chengyan Wang Luping Ma Hao Peng Xingxing Dong Jinfang Jiang Xianling Lu Lijuan Pang 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
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: Mol Biol Cell - September 6, 2023 Category: Molecular Biology Authors: Mingxia Wang Menghui Chen Xia Wu Xinbo Huang Bo Yu Source Type: research
Scale-Up of Protein Purification: Downstream Processing Issues
Methods Mol Biol. 2023;2699:61-75. doi: 10.1007/978-1-0716-3362-5_5.ABSTRACTLarge-scale chromatography operations continue to occupy the central position in the overall strategy for downstream processing and purification of therapeutic protein products for human use. As the biopharmaceutical industry looks forward to embracing new therapeutic modalities such as viral vector-mediated gene therapy, it is becoming evident that chromatographic separations will be also be crucial for success in that discipline. The current industry focus on cell culture intensification strategies that can result in increased process efficiency ...
Source: Mol Biol Cell - August 30, 2023 Category: Molecular Biology Authors: John Joseph Milne Source Type: research
Chromatographic Purification of Viral Vectors for Gene Therapy Applications
Methods Mol Biol. 2023;2699:51-60. doi: 10.1007/978-1-0716-3362-5_4.ABSTRACTChromatography has been a mainstay in the downstream processing and purification of biopharmaceutical medicines. Until now, this has largely involved the purification of protein products such as recombinant enzymes and monoclonal antibodies using large-scale column chromatography methods. The development of advanced therapeutic medicinal products (ATMP) is heralding in a new era of therapeutics for a range of indications. These new therapeutics use diverse substances ranging from live stem cell preparations to fragments of nucleic acid enclosed in ...
Source: Mol Biol Cell - August 30, 2023 Category: Molecular Biology Authors: Aoife Mair éad Kearney Source Type: research
Scale-Up of Protein Purification: Downstream Processing Issues
Methods Mol Biol. 2023;2699:61-75. doi: 10.1007/978-1-0716-3362-5_5.ABSTRACTLarge-scale chromatography operations continue to occupy the central position in the overall strategy for downstream processing and purification of therapeutic protein products for human use. As the biopharmaceutical industry looks forward to embracing new therapeutic modalities such as viral vector-mediated gene therapy, it is becoming evident that chromatographic separations will be also be crucial for success in that discipline. The current industry focus on cell culture intensification strategies that can result in increased process efficiency ...
Source: Mol Biol Cell - August 30, 2023 Category: Molecular Biology Authors: John Joseph Milne Source Type: research
Chromatographic Purification of Viral Vectors for Gene Therapy Applications
Methods Mol Biol. 2023;2699:51-60. doi: 10.1007/978-1-0716-3362-5_4.ABSTRACTChromatography has been a mainstay in the downstream processing and purification of biopharmaceutical medicines. Until now, this has largely involved the purification of protein products such as recombinant enzymes and monoclonal antibodies using large-scale column chromatography methods. The development of advanced therapeutic medicinal products (ATMP) is heralding in a new era of therapeutics for a range of indications. These new therapeutics use diverse substances ranging from live stem cell preparations to fragments of nucleic acid enclosed in ...
Source: Mol Biol Cell - August 30, 2023 Category: Molecular Biology Authors: Aoife Mair éad Kearney Source Type: research
Checkpoint blockade meets gene therapy: Opportunities to improve response and reduce toxicity
Int Rev Cell Mol Biol. 2023;379:43-86. doi: 10.1016/bs.ircmb.2023.05.006. Epub 2023 Jun 23.ABSTRACTImmune checkpoint inhibitors (ICIs) based on monoclonal antibodies represent a breakthrough for the treatment of cancer. However, their efficacy varies among tumor types and patients, and they can lead to adverse effects due to on-target/off-tumor activity, since they are administered systemically at high doses. An alternative and attractive approach for the delivery of ICIs is the use of gene therapy vectors able to express them in vivo. This review focuses on the most recent studies using viral vectors able to express ICIs ...
Source: Mol Biol Cell - August 4, 2023 Category: Molecular Biology Authors: Noelia Silva-Pilipich Ángela Covo-Vergara Luc ía Vanrell Cristian Smerdou Source Type: research
Viral vectors engineered for gene therapy
Int Rev Cell Mol Biol. 2023;379:1-41. doi: 10.1016/bs.ircmb.2023.05.005. Epub 2023 Jul 1.ABSTRACTGene therapy has seen major progress in recent years. Viral vectors have made a significant contribution through efficient engineering for improved delivery and safety. A large variety of indications such as cancer, cardiovascular, metabolic, hematological, neurological, muscular, ophthalmological, infectious diseases, and immunodeficiency have been targeted. Viral vectors based on adenoviruses, adeno-associated viruses, herpes simplex viruses, retroviruses including lentiviruses, alphaviruses, flaviviruses, measles viruses, rh...
Source: Mol Biol Cell - August 4, 2023 Category: Molecular Biology Authors: Kenneth Lundstrom Source Type: research
AAV Serotypes and Their Suitability for Retinal Gene Therapy
Adv Exp Med Biol. 2023;1415:131-134. doi: 10.1007/978-3-031-27681-1_20.ABSTRACTThroughout the last 25 years, exceptional progress in retinal gene therapy was achieved. The major breakthrough was realized in 2017 when the FDA approved the adeno-associated virus (AAV)-based gene therapy for treatment of the monogenetic disorder Leber congenital amaurosis type 2 (LCA2). Since then, many therapies for inherited retinal diseases (IRD) reached phase I/II clinical trials, targeting diseases like achromatopsia, choroideremia, retinitis pigmentosa, Stargardt disease, and many more (reviewed in (Trapani and Auricchio, Trends Mol Med...
Source: Mol Biol Cell - July 13, 2023 Category: Molecular Biology Authors: Lynn J A Ebner Christian Grimm Source Type: research
Detection of Single Non-small Cell Lung Cancer Cell Multidrug Resistance with Single-Cell Bioanalyzer
Methods Mol Biol. 2023;2689:1-11. doi: 10.1007/978-1-0716-3323-6_1.ABSTRACTNon-small cell lung cancer (NSCLC) is one of the leading causes of cancer death in the world. Despite the development of various lung cancer treatment methods, including surgery, radiation therapy, endocrine therapy, immunotherapy, and gene therapy, chemotherapy remains the most common approach for treating cancer. The risk of tumors acquiring resistance to chemotherapy remains a significant hurdle to the use of this approach for the successful treatment of various types of cancer. The majority of cancer-related deaths are related to metastasis. Cir...
Source: Mol Biol Cell - July 10, 2023 Category: Molecular Biology Authors: Jun Cai Ya-Bing Cao Elaine Lai-Han Leung Source Type: research
CKM and TERT dual promoters drive CRISPR-dCas9 to specifically inhibit the malignant behavior of osteosarcoma cells
Cell Mol Biol Lett. 2023 Jul 6;28(1):52. doi: 10.1186/s11658-023-00464-7.ABSTRACTImprovements in treatment and chemotherapy have increased the survival rate of osteosarcoma, but overall efficacy remains low, highlighting the need for new gene therapy methods. Clustered regularly interspaced short palindromic repeats-deactivated Cas9 (CRISPR-dCas9) technology offers a promising strategy, but targeting osteosarcoma cells precisely is a challenge. We designed a system to achieve specific expression of CRISPR-dCas9-KRAB in osteosarcoma cells by using the creatine kinase muscle (CKM) promoter to drive dCas9-KRAB and the telomer...
Source: Mol Biol Cell - July 6, 2023 Category: Molecular Biology Authors: Yawei Hu Hao Zhang Zengfeng Guo Jianhua Zhou Wang Zhang Ming Gong Jiawen Wu Source Type: research
