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Physical Characteristics and Stability Profile of Recombinant Plasmid DNA within a Film Matrix
The objective of these studies was to identify a film-based formulation that preserved transfection efficiency of plasmids at 25°C. A model plasmid, pAAVlacZ, was used for these studies. Transfection efficiency and agarose gel electrophoresis were utilized to assess bioactivity and changes in physical conformation of plasmid during storage. An amino acid, capable of sustaining a positive charge while supporting an alkaline environment within the film matrix, preserved transfection efficiency for 9 months at 25°C. Addition of sugar and a plasticizer to the formulation preserved the plasmid in an amorphous state and improv...
Source: European Journal of Pharmaceutics and Biopharmaceutics - August 11, 2023 Category: Drugs & Pharmacology Authors: Trang Nguyen Kieu Doan Maria A Croyle Source Type: research
Thermodynamic and cellular studies of doxorubicin/daunorubicin loaded by a DNA tetrahedron for diagnostic imaging, chemotherapy, and gene therapy
In this study, we designed an antisense oligonucleotides (ASOs)-linked DNA tetrahedron (ASOs-TD). The detection limit of ASOs-TD for MDR1 mRNA was 0.05 μM. By using fluorescence spectroscopy and isothermal titration calorimetry (ITC), the interactions between doxorubicin (DOX) /daunorubicin (DAU) and ASOs-TD were investigated. The number of binding sites (n), binding constant (Ka), entropy change (ΔSo), enthalpy change (ΔHo) and Gibbs free energy change (ΔGo) were obtained. The intercalation of DOX/DAU with ASOs-TD was demonstrated by differential scanning calorimetry (DSC) and quenching researches of potassium ferricy...
Source: International Journal of Biological Macromolecules - August 10, 2023 Category: Biochemistry Authors: Xinyu Li Yushu Wu Xinpeng Zhang Jie Liu Yanqing Zhang Lixia Yuan Min Liu Source Type: research
Emerging 2D pnictogens: a novel multifunctional photonic nanoplatform for cutting-edge precision treatment
Chem Commun (Camb). 2023 Aug 9. doi: 10.1039/d3cc02624h. Online ahead of print.ABSTRACTThe elements of the pnictogen group, known as the 15th (VA) family in the periodic table, including phosphorus (P), arsenic (As), antimony (Sb) and bismuth (Bi), have been widely used by alchemists to treat various diseases since ancient times and hold a pivotal position in the history of medicine, owing to their diverse pharmacological activities. Recently, with the development of modern nanotechnology, pnictogen group elements appear in a more innovative form, namely two-dimensional (2D) pnictogens (i.e. phosphorene, arsenene, and bism...
Source: Chemical Communications - August 9, 2023 Category: Chemistry Authors: Wenjing Zheng Yifan Zhang Ming Gao Meng Qiu Source Type: research
Platelet-targeted gene therapy induces immune tolerance in hemophilia and beyond
J Thromb Haemost. 2023 Aug 7:S1538-7836(23)00589-5. doi: 10.1016/j.jtha.2023.07.025. Online ahead of print.ABSTRACTBlood platelets have unique storage and delivery capabilities. Platelets play fundamental roles in hemostasis, inflammatory reactions, and immune responses. Beyond their functions, platelets have been used as a target for gene therapy. Platelet-targeted gene therapy aims to deliver a sustained expression of neo-protein in vivo by genetically modifying the target cells, resulting in a cure for the disease. Even though there has been substantial progress in the field of gene therapy, the potential development of...
Source: Thrombosis and Haemostasis - August 9, 2023 Category: Hematology Authors: Saurabh Kumar Jocelyn A Schroeder Qizhen Shi Source Type: research
Genetic testing and gene therapy in retinal diseases: Knowledge and perceptions of optometrists in Australia and New Zealand
This study found that optometrists in Australia and New Zealand have a high level of interest in ocular genetics topics. However, knowledge gaps include referral pathways and awareness of genetic testing and gene therapy outcomes. Addressing perceived barriers to access and promoting sharing of knowledge between interdisciplinary networks can set the foundation for genetic education agendas in primary eye care.PMID:37553298 | DOI:10.1111/cge.14415
Source: Clinical Genetics - August 8, 2023 Category: Genetics & Stem Cells Authors: Alexis Ceecee Britten-Jones Heather G Mack Andrea L Vincent Lisa J Hill Thomas L Edwards Lauren N Ayton Source Type: research
Gene therapy of adeno-associated virus (AAV) vectors in preclinical models of ischemic stroke
CNS Neurosci Ther. 2023 Aug 8. doi: 10.1111/cns.14392. Online ahead of print.ABSTRACTStroke has been associated with devastating clinical outcomes, with current treatment strategies proving largely ineffective. Therefore, there is a need to explore alternative treatment options for addressing post-stroke functional deficits. Gene therapy utilizing adeno-associated viruses (AAVs) as a critical gene vector delivering genes to the central nervous system (CNS) gene delivery has emerged as a promising approach for treating various CNS diseases. This review aims to provide an overview of the biological characteristics of AAV vec...
Source: CNS Neuroscience and Therapeutics - August 8, 2023 Category: Neuroscience Authors: Jing Wang Mengna Zhu Jingyi Sun Lina Feng Mingfeng Yang Baoliang Sun Leilei Mao Source Type: research
Natural deep eutectic solvents protect RNA from thermal-induced degradation
Arch Biochem Biophys. 2023 Aug 5:109714. doi: 10.1016/j.abb.2023.109714. Online ahead of print.ABSTRACTRNA is a fundamental nucleic acid for life and it plays important roles in the regulation of gene transcription, post-transcriptional regulation, and epigenetic regulation. Recently, the focus on this nucleic acid has significantly increased due to the development of mRNA vaccines and RNA-based gene therapy protocols. Unfortunately, RNA based products show constrains mainly owing to instability and easy degradability of the RNA molecules. Indeed, unlike the DNA molecule which has a great intrinsic stability, RNA is more p...
Source: Archives of Biochemistry and Biophysics - August 7, 2023 Category: Biochemistry Authors: Roberto Mattioli Anna Maggiore Daniel Di Risola Rodolfo Federico Maria D'Erme Antonio Francioso Luciana Mosca Source Type: research
Clinical Efficacy and Safety of AdV-tk Gene Therapy for Patients with Cervical Squamous Intraepithelial Lesion: A Prospective Study
Hum Gene Ther. 2023 Aug 5. doi: 10.1089/hum.2023.066. Online ahead of print.ABSTRACTCervical cancer is the fourth most common type of cancer for women in 2020, and many more women have cervical pre-cancerous lesion-squamous intraepithelial lesion (SIL). Early treatment of cervical SIL to reverse or delay its progression is an important approach to reduce the incidence of cervical cancer. The efficacy and safety of adenovirus-based vectors expressing the thymidine kinase gene (AdV-tk) in the treatment of multiple types of cancers shows promise for its use in gynecology. We aim to provide relevant clinical efficacy and safet...
Source: Adv Data - August 5, 2023 Category: Epidemiology Authors: Nan Wang Xiulan Li Xin Liu Meina Bian Ying Hou Yuxiang Zhou Yanmei Li Fuqiang Xu Huadong Tang Ning Li Qing Liu Source Type: research
Production of retroviral vectors in continuous high cell density culture
Appl Microbiol Biotechnol. 2023 Aug 5. doi: 10.1007/s00253-023-12689-9. Online ahead of print.ABSTRACTRetroviral vectors derived from murine leukemia virus (MLV) are used in somatic gene therapy applications e.g. for genetic modification of hematopoietic stem cells. Recently, we reported on the establishment of a suspension viral packaging cell line (VPC) for the production of MLV vectors. Human embryonic kidney 293-F (HEK293-F) cells were genetically modified for this purpose using transposon vector technology. Here, we demonstrate the establishment of a continuous high cell density (HCD) process using this cell line. Fir...
Source: Applied Microbiology and Biotechnology - August 5, 2023 Category: Microbiology Authors: Marc D Hein Daniel Kazenmaier Yasemin van Heuvel Tanya Dogra Maurizio Cattaneo Sascha Y Kupke J örn Stitz Yvonne Genzel Udo Reichl Source Type: research
Clinical Efficacy and Safety of AdV-tk Gene Therapy for Patients with Cervical Squamous Intraepithelial Lesion: A Prospective Study
Hum Gene Ther. 2023 Aug 5. doi: 10.1089/hum.2023.066. Online ahead of print.ABSTRACTCervical cancer is the fourth most common type of cancer for women in 2020, and many more women have cervical pre-cancerous lesion-squamous intraepithelial lesion (SIL). Early treatment of cervical SIL to reverse or delay its progression is an important approach to reduce the incidence of cervical cancer. The efficacy and safety of adenovirus-based vectors expressing the thymidine kinase gene (AdV-tk) in the treatment of multiple types of cancers shows promise for its use in gynecology. We aim to provide relevant clinical efficacy and safet...
Source: Adv Data - August 5, 2023 Category: Epidemiology Authors: Nan Wang Xiulan Li Xin Liu Meina Bian Ying Hou Yuxiang Zhou Yanmei Li Fuqiang Xu Huadong Tang Ning Li Qing Liu Source Type: research
Production of retroviral vectors in continuous high cell density culture
Appl Microbiol Biotechnol. 2023 Aug 5. doi: 10.1007/s00253-023-12689-9. Online ahead of print.ABSTRACTRetroviral vectors derived from murine leukemia virus (MLV) are used in somatic gene therapy applications e.g. for genetic modification of hematopoietic stem cells. Recently, we reported on the establishment of a suspension viral packaging cell line (VPC) for the production of MLV vectors. Human embryonic kidney 293-F (HEK293-F) cells were genetically modified for this purpose using transposon vector technology. Here, we demonstrate the establishment of a continuous high cell density (HCD) process using this cell line. Fir...
Source: Applied Microbiology and Biotechnology - August 5, 2023 Category: Microbiology Authors: Marc D Hein Daniel Kazenmaier Yasemin van Heuvel Tanya Dogra Maurizio Cattaneo Sascha Y Kupke J örn Stitz Yvonne Genzel Udo Reichl 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: International Review of Cell and Molecular Biology - August 4, 2023 Category: Cytology Authors: Noelia Silva-Pilipich Ángela Covo-Vergara Luc ía Vanrell Cristian Smerdou Source Type: research
