Journal of Chromatography A, Год журнала: 2025, Номер 1748, С. 465832 - 465832
Опубликована: Март 2, 2025
Язык: Английский
Advanced Materials, Год журнала: 2024, Номер 36(31)
Опубликована: Июнь 6, 2024
Abstract The recent success of gene therapy during the COVID‐19 pandemic has underscored importance effective and safe delivery systems. Complementing lipid‐based systems, polymers present a promising alternative for delivery. Significant advances have been made in past, with multiple clinical trials progressing beyond phase I several companies actively working on polymeric systems which provides assurance that carriers can soon achieve translation. massive advantage structural tunability vast chemical space is being leveraged to mitigate shortcomings traditional polycationic improve translatability Tailored approaches diverse nucleic acids specific subcellular targets are now designed therapeutic efficacy. This review describes polymer design improved by polyplexes covalent polymer‐nucleic acid conjugates. also offers brief note novel computational techniques design. concludes an overview current state therapies clinic as well future directions their translation clinic.
Язык: Английский
Процитировано
17
The Journal of Gene Medicine, Год журнала: 2024, Номер 26(8)
Опубликована: Авг. 1, 2024
Abstract To date, 3,900 gene therapy clinical trials have been completed, are ongoing or approved worldwide. Our database brings together global information on activity from trial databases, official agency sources, published literature, conference presentations and posters kindly provided to us by individual investigators sponsors. This review presents our analysis of that, the best knowledge, being performed As March 2023 update, we entries undertaken in 46 countries. We analyzed geographical distribution trials, disease indications (or other reasons) for proportions which different vector types used, genes transferred. Details analyses presented, searchable The Journal Gene Medicine Therapy Clinical Trials Worldwide website at https://a873679.fmphost.com/fmi/webd/GTCT . also provide an overview progress made around world, discuss key trends since previous review, namely unprecedented increase activity, including implementation genome editing technology with potential transform field moving forward.
Язык: Английский
Процитировано
17
Journal of Chromatography A, Год журнала: 2024, Номер 1716, С. 464632 - 464632
Опубликована: Янв. 9, 2024
Язык: Английский
Процитировано
13
Nature Biotechnology, Год журнала: 2025, Номер unknown
Опубликована: Янв. 29, 2025
Abstract Therapeutic efficacy and safety of adeno-associated virus (AAV) liver gene therapy depend on capsid choice. To predict AAV performance under near-clinical conditions, we established side-by-side comparison at single-cell resolution in human livers maintained by normothermic machine perfusion. AAV-LK03 transduced hepatocytes much more efficiently specifically than AAV5, AAV8 AAV6, which are most commonly used clinically, AAV-NP59, is better transducing engrafted immune-deficient mice. preferentially periportal normal liver, whereas AAV5 targeted pericentral steatotic liver. sinusoidal endothelial cells as hepatocytes. steatosis influenced vector episome formation, determines durability, with delaying concatemerization. Our findings inform choice clinical therapy, including consideration disease-relevant hepatocyte zonation effects steatosis, facilitate the development capsids that transduce or other therapeutically relevant cell types maximum efficiency specificity.
Язык: Английский
Процитировано
2
Journal of Chromatography A, Год журнала: 2025, Номер 1748, С. 465832 - 465832
Опубликована: Март 2, 2025
Язык: Английский
Процитировано
2