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REVIEW
Masukazu Inoie
Japan Tissue Engineering Co., Ltd., J-TEC, was launched in 1999 to industrialize regenerative medicine in Japan. We developed the first regenerative medicine product, JACE (autologous cultured epidermis), which received PMDA approval for treating serious burns in 2007. Then, JACC (autologous cultured cartilage), the second product, was approved in 2012 for efficacy on traumatic cartilage defects. In 2014, the Pharmaceutical Affairs Law was revised to the Pharmaceutical and Medical Device Act, and regenerative medicine products, including gene therapies, were newly classified to accelerate productization...
2024: Nihon Yakurigaku Zasshi. Folia Pharmacologica Japonica
#22
REVIEW
Kahori Shimizu
The number of patients with lifestyle-related diseases such as type 2 diabetes mellitus (T2DM) and metabolic dysfunction-associated steatotic liver disease (MASLD), formerly known as non-alcoholic fatty liver disease (NAFLD), has continued to increase worldwide. Therefore, development of innovative therapeutic methods targeting lifestyle-related diseases is required. Gene therapy has attracted considerable attention as an advanced medical treatment. Safe and high-performance vectors are essential for the practical application of gene therapy...
2024: Biological & Pharmaceutical Bulletin
#23
REVIEW
David Catalán-Tatjer, Konstantina Tzimou, Lars K Nielsen, Jesús Lavado-García
Recombinant adeno-associated viruses (rAAVs) stand at the forefront of gene therapy applications, holding immense significance for their safe and efficient gene delivery capabilities. The constantly increasing and unmet demand for rAAVs underscores the need for a more comprehensive understanding of AAV biology and its impact on rAAV production. In this literature review, we delved into AAV biology and rAAV manufacturing bioprocesses, unravelling the functions and essentiality of proteins involved in rAAV production...
April 29, 2024: Biotechnology Advances
#24
JOURNAL ARTICLE
Ahmed A H Abdellatif, Abdellatif Bouazzaoui, Hesham M Tawfeek, Mahmoud A Younis
Breast cancer is a wide-spread threat to the women's health. The drawbacks of conventional treatments necessitate the development of alternative strategies, where gene therapy has regained hope in achieving an efficient eradication of aggressive tumors. Monocarboxylate transporter 4 (MCT4) plays pivotal roles in the growth and survival of various tumors, which offers a promising target for treatment. In the present study, pH-responsive lipid nanoparticles (LNPs) based on the ionizable lipid,1,2-dioleoyl-3-dimethylammonium propane (DODAP), were designed for the delivery of siRNA targeting MCT4 gene to the breast cancer cells...
April 25, 2024: Colloids and Surfaces. B, Biointerfaces
#25
JOURNAL ARTICLE
Giulia Consiglieri, Francesca Tucci, Maurizio De Pellegrin, Barbara Guerrini, Alessandro Cattoni, Giulia Risca, Stefano Scarparo, Marina Sarzana, Silvia Pontesilli, Renata Mellone, Serena Gasperini, Stefania Galimberti, Paolo Silvani, Chiara Filisetti, Silvia Darin, Giulia Forni, Simona Miglietta, Ludovica Santi, Marcella Facchini, Ambra Corti, Francesca Fumagalli, Maria Pia Cicalese, Valeria Calbi, Maddalena Migliavacca, Federica Barzaghi, Francesca Ferrua, Vera Gallo, Salvatore Recupero, Daniele Canarutto, Matteo Doglio, Lucia Tedesco, Nicola Volpi, Attilio Rovelli, Giancarlo la Marca, Maria Grazia Valsecchi, Stefano Zancan, Fabio Ciceri, Luigi Naldini, Cristina Baldoli, Rossella Parini, Bernhard Gentner, Alessandro Aiuti, Maria Ester Bernardo
Mucopolysaccharidosis type I Hurler (MPSIH) is characterized by severe and progressive skeletal dysplasia that is not fully addressed by allogeneic hematopoietic stem cell transplantation (HSCT). Autologous hematopoietic stem progenitor cell-gene therapy (HSPC-GT) provides superior metabolic correction in patients with MPSIH compared with HSCT; however, its ability to affect skeletal manifestations is unknown. Eight patients with MPSIH (mean age at treatment: 1.9 years) received lentiviral-based HSPC-GT in a phase 1/2 clinical trial (NCT03488394)...
May 2024: Science Translational Medicine
#26
JOURNAL ARTICLE
David M Rind
No abstract text is available yet for this article.
May 1, 2024: JAMA
#27
JOURNAL ARTICLE
Xin Xu, Ni Han, Fangkun Zhao, Ruoyue Fan, Qingguo Guo, Xuefei Han, Ying Liu, Guangzuo Luo
Retinal neovascularization (RNV) is primarily driven by vascular endothelial growth factor (VEGF). However, current anti-VEGF therapies are limited by short half-lives and repeated injections, which reduce patient quality of life and increase medical risks. Additionally, not all patients benefit from anti-VEGF monotherapy, and some problems, such as unsatisfactory vision recovery, persist after long-term treatment. In this study, we constructed a recombinant adeno-associated virus (AAV), AAV2-SPLTH, which encodes an anti-VEGF antibody similar to bevacizumab, and assessed its effects in a doxycycline-induced Tet-opsin-VEGFA mouse model of RNV...
June 11, 2024: Molecular Therapy. Nucleic Acids
#28
JOURNAL ARTICLE
Daniela Cesana, Maria Pia Cicalese, Andrea Calabria, Pietro Merli, Roberta Caruso, Monica Volpin, Laura Rudilosso, Maddalena Migliavacca, Federica Barzaghi, Claudia Fossati, Francesco Gazzo, Simone Pizzi, Andrea Ciolfi, Alessandro Bruselles, Francesca Tucci, Giulio Spinozzi, Giulia Pais, Fabrizio Benedicenti, Matteo Barcella, Ivan Merelli, Pierangela Gallina, Stefania Giannelli, Francesca Dionisio, Serena Scala, Miriam Casiraghi, Luisa Strocchio, Luciana Vinti, Lucia Pacillo, Eleonora Draghi, Marcella Cesana, Sara Riccardo, Chiara Colantuono, Emmanuelle Six, Marina Cavazzana, Filippo Carlucci, Manfred Schmidt, Caterina Cancrini, Fabio Ciceri, Luca Vago, Davide Cacchiarelli, Bernhard Gentner, Luigi Naldini, Marco Tartaglia, Eugenio Montini, Franco Locatelli, Alessandro Aiuti
Hematopoietic stem cell gene therapy (GT) using a γ-retroviral vector (γ-RV) is an effective treatment for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency. Here, we describe a case of GT-related T-cell acute lymphoblastic leukemia (T-ALL) that developed 4.7 years after treatment. The patient underwent chemotherapy and haploidentical transplantation and is currently in remission. Blast cells contain a single vector insertion activating the LIM-only protein 2 (LMO2) proto-oncogene, confirmed by physical interaction, and low Adenosine Deaminase (ADA) activity resulting from methylation of viral promoter...
April 30, 2024: Nature Communications
#29
JOURNAL ARTICLE
Xu-Rui Gu, Yifan Tai, Zhen Liu, Xinyan Zhang, Kun Liu, Ling-Yun Zhou, Wen-Jun Yin, Yi-Xuan Deng, Deling Kong, Adam C Midgley, Xiao-Cong Zuo
The mitochondrial enzyme arginase-2 (Arg-2) is implicated in the pathophysiology of contrast-induced acute kidney injury (CI-AKI). Therefore, Arg-2 represents a candid target for CI-AKI prevention. Here, we developed layer-by-layer (LbL) assembled renal-targeting polymeric nanoparticles to efficiently deliver small interfering RNA (siRNA), knockdown Arg-2 expression in renal tubules, and evaluated prevention of CI-AKI. Firstly, near-infrared dye-loaded poly(lactic-co-glycolic acid) (PLGA) anionic cores were electrostatically-coated with cationic chitosan (CS) to facilitate the adsorption and stabilization of Arg-2 siRNA...
April 30, 2024: Advanced Healthcare Materials
#30
JOURNAL ARTICLE
Kiril K Poukalov, M Carmen Valero, Derek R Muscato, Leanne M Adams, Heejae Chun, Young Il Lee, Nadja S Andrade, Zane Zeier, H Lee Sweeney, Eric T Wang
Successful CRISPR/Cas9-based gene editing in skeletal muscle is dependent on efficient propagation of Cas9 to all myonuclei in the myofiber. However, nuclear-targeted gene therapy cargos are strongly restricted to their myonuclear domain of origin. By screening nuclear localization signals and nuclear export signals, we identify "Myospreader," a combination of short peptide sequences that promotes myonuclear propagation. Appending Myospreader to Cas9 enhances protein stability and myonuclear propagation in myoblasts and myofibers...
May 7, 2024: Proceedings of the National Academy of Sciences of the United States of America
#31
REVIEW
Hyun-Ju Lee, Hee-Jeong Choi, Yoo Joo Jeong, Yoon-Hee Na, Jin Tae Hong, Ji Min Han, Hyang-Sook Hoe, Key-Hwan Lim
The prevalence of Alzheimer's disease (AD) and its associated economic and societal burdens are on the rise, but there are no curative treatments for AD. Interestingly, this neurodegenerative disease shares several biological and pathophysiological features with cancer, including cell-cycle dysregulation, angiogenesis, mitochondrial dysfunction, protein misfolding, and DNA damage. However, the genetic factors contributing to the overlap in biological processes between cancer and AD have not been actively studied...
April 27, 2024: International Journal of Biological Macromolecules
#32
JOURNAL ARTICLE
Michela Milani, Cesare Canepari, Simone Assanelli, Simone Merlin, Ester Borroni, Francesco Starinieri, Mauro Biffi, Fabio Russo, Anna Fabiano, Desirèe Zambroni, Andrea Annoni, Luigi Naldini, Antonia Follenzi, Alessio Cantore
Lentiviral vectors (LV) are efficient vehicles for in vivo gene delivery to the liver. LV integration into the chromatin of target cells ensures their transmission upon proliferation, thus allowing potentially life-long gene therapy following a single administration, even to young individuals. The glycoprotein of the vesicular stomatitis virus (VSV.G) is widely used to pseudotype LV, as it confers broad tropism and high stability. The baculovirus-derived GP64 envelope protein has been proposed as an alternative for in vivo liver-directed gene therapy...
April 29, 2024: EMBO Molecular Medicine
#33
JOURNAL ARTICLE
Alexander Renner, Anika Stahringer, Katharina Eva Ruppel, Stephan Fricke, Ulrike Koehl, Dominik Schmiedel
Allogeneic cell therapies, such as those involving macrophages or Natural Killer (NK) cells, are of increasing interest for cancer immunotherapy. However, the current techniques for genetically modifying these cell types using lenti- or gamma-retroviral vectors present challenges, such as required cell pre-activation and inefficiency in transduction, which hinder the assessment of preclinical efficacy and clinical translation. In our study, we describe a novel lentiviral pseudotype based on the Koala Retrovirus (KoRV) envelope protein, which we identified based on homology to existing pseudotypes used in cell therapy...
April 29, 2024: Gene Therapy
#34
JOURNAL ARTICLE
William L Herring, Meghan E Gallagher, Nirmish Shah, K C Morse, Deirdre Mladsi, Olivia M Dong, Anjulika Chawla, Jennifer W Leiding, Lixin Zhang, Clark Paramore, Biree Andemariam
BACKGROUND AND OBJECTIVE: Gene therapies for sickle cell disease (SCD) may offer meaningful benefits for patients and society. This study evaluated the cost-effectiveness of lovotibeglogene autotemcel (lovo-cel), a one-time gene therapy administered via autologous hematopoietic stem cell transplantation, compared with common care for patients in the United States (US) with SCD aged ≥ 12 years with ≥ 4 vaso-occlusive events (VOEs) in the past 24 months...
April 29, 2024: PharmacoEconomics
#35
JOURNAL ARTICLE
Suresh Agarwal, Cedric Hermans, Wolfgang Miesbach, Flora Peyvandi, Robert Sidonio, Dane Osmond, Vanessa Newman, Josh Henshaw, Steven Pipe
INTRODUCTION: Valoctocogene roxaparvovec, a gene therapy evaluated in the phase 3 GENEr8-1 trial, supports endogenous factor VIII (FVIII) production to prevent bleeding in people with severe haemophilia A. Individuals receiving emicizumab, an antibody mimicking the function of activated FVIII, were excluded from GENEr8-1 enrolment since emicizumab was an investigational therapy at the time of trial initiation. AIM: Utilize pharmacokinetic simulations to provide guidance on best practices for maintaining haemostatic control while transitioning from emicizumab prophylaxis to valoctocogene roxaparvovec...
April 29, 2024: Haemophilia: the Official Journal of the World Federation of Hemophilia
#36
JOURNAL ARTICLE
Curtis W Jarand, Karen Baker, Matthew Petroff, Mi Jin, Wayne F Reed
While adeno-associated virus is a leading vector for gene therapy, significant gaps remain in understanding AAV degradation and stability. In this work, we study the degradation of an engineered AAV serotype at physiological pH and ionic strength. Viral particles of varying fractions of encapsulated DNA were incubated between 30 and 60 °C, with changes in molecular weight measured by changes in total light scattering intensity at 90° over time. Mostly full vectors demonstrated a rapid decrease in molecular weight corresponding to the release of capsid DNA, followed by slow aggregation...
April 29, 2024: Biomacromolecules
#37
JOURNAL ARTICLE
Dujuan Li, Yuxin Lu, Fengjun Xiao, Xiaochen Cheng, Chunsheng Hu, Xuefeng Zhu, Xiaoying Wang, Haiying Duan, Li Du, Qinglin Zhang
Combined radiation-trauma skin injury represents a severe and intractable condition that urgently requires effective therapeutic interventions. In this context, hepatocyte growth factor (HGF), a multifunctional growth factor with regulating cell survival, angiogenesis, anti-inflammation and antioxidation, may be valuable for the treatment of combined radiation-trauma injury. This study investigated the protective effects of a recombinant plasmid encoding human HGF (pHGF) on irradiated human immortalized keratinocytes (HaCaT) cells in vitro, and its capability to promote the healing of combined radiation-trauma injuries in mice...
April 28, 2024: Journal of Radiation Research
#38
JOURNAL ARTICLE
Rajani Knanghat, Sanjib Senapati
Deoxyribonucleic acid (DNA) stability is a prerequisite in many applications, ranging from DNA-based vaccines and data storage to gene therapy. However, the strategies to enhance DNA stability are limited, and the underlying mechanisms are poorly understood. Ionic liquids (ILs), molten salts of organic cations and organic/inorganic anions, are showing tremendous prospects in myriads of applications. With a judicious choice of constituent ions, the protic nature of ILs can be tuned. In this work, we investigate the relative stability of full-length genomic DNA in aqueous IL solutions of increasing protic nature...
April 29, 2024: Journal of Physical Chemistry. B
#39
JOURNAL ARTICLE
Tianming Jiang, Jie Zheng, Nana Li, Xiaodong Li, Jixing He, Junde Zhou, Boshi Sun, Qiang Chi
INTRODUCTION: Crohn's disease (CD) and colorectal cancer (CRC) represent a group of intestinal disorders characterized by intricate pathogenic mechanisms linked to the disruption of intestinal immune homeostasis. Therefore, comprehending the immune response mechanisms in both categories of intestinal disorders is of paramount significance in the prevention and treatment of these debilitating intestinal ailments. METHOD: IIn this study, we conducted single-cell analysis on paired samples obtained from primary colorectal tumors and individuals with Crohn's disease, which was aimed at deciphering the factors influencing the composition of the intestinal immune microenvironment...
February 6, 2024: Current Gene Therapy
#40
REVIEW
Runxuan Chu, Yi Wang, Jianglong Kong, Ting Pan, Yani Yang, Jun He
The liver, a complex and vital organ in the human body, is susceptible to various diseases, including metabolic disorders, acute hepatitis, cirrhosis, and hepatocellular carcinoma. In recent decades, these diseases have significantly contributed to global morbidity and mortality. Currently, liver transplantation remains the most effective treatment for hepatic disorders. Nucleic acid therapeutics offer a selective approach to disease treatment through diverse mechanisms, enabling the regulation of relevant genes and providing a novel therapeutic avenue for hepatic disorders...
April 29, 2024: Journal of Materials Chemistry. B, Materials for Biology and Medicine
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