Self-amplifying RNA, circular RNA, CRISPR systems, antisense oligonucleotides, and antibody-oligonucleotide conjugates are presented, as well as CAR modification of T-cells, natural killer cells, and macrophages, with a brief discussion of modified stem cells.
Abstract
Recent advancements in experimental and computational methods along with growing gene sequencing libraries have enabled rapid development of nucleic acid-based therapeutic design for a wide variety of diseases. New drugs, delivery tools and methods have enabled precise and efficient therapy at a molecular scale. This review covers the current landscape and future directions of methods for gene and cell therapies, as well as their applications, advantages, and limitations. We present self-amplifying RNA, circular RNA, CRISPR systems, antisense oligonucleotides, and antibody-oligonucleotide conjugates, as well as CAR modification of T-cells, natural killer cells, and macrophages, with a brief discussion of modified stem cells. We also discuss developments in viral and non-viral delivery systems for their applications in cell and gene therapies.
This article provides a comprehensive overview of the evolving landscape of nucleic acid-based therapeutics and aims to inform clinicians, researchers, and pharmaceutical scientists about their current applications and future potential in modern healthcare.
Satyaraj Ombase, Shanmugarathanam Alagarsamy, R. Prasad et al.· Adolescência e Saúde· 0 citations
The different forms of RNA-based treat-ments, such as messenger RNAs, small interfering RNAs, and circular RNAs, are discussed in this paper along with their significance in gene regulation and the treatment of disease.
Avinash Verma, Shaweta Sharma· Drug Delivery Letters· 0 citations
This review starts by examining the advantages and limitations of various physical methods for delivering Cas9, and highlights key applications of CRISPR systems in epigenetic modifications, and explores the use of CRISPR-Cas9 technology in genome editing, with a particular focus on base editing and prime editing.
Suhail Ahmad Sheikh, W. Shah, Azka Khan et al.· Journal of Population Therap...· 0 citations
Gene therapy has found wide use in the prevention and treatment of cancer, infectious diseases, and neurodegenerative and metabolic disorders. Meanwhile, the development of formulations providing effective and targeted delivery of nucleic acids has been and remains a challenging interdisciplinary task. Polymeric system...
E. Shaputkin, I. Nifant'ev, P. Ivchenko· Russian Chemical Reviews· 0 citations
This review provides a comprehensive overview of the current status of viral and non-viral vector systems for in vivo and ex vivo applications, and key comparisons are made across safety, efficacy, scalability, and immune responses.
Proteins and nucleic acids represent nature’s most versatile biomolecules. By leveraging the functionality of proteins and the programmability of nucleic acids, along with the exquisite recognition properties of both, protein-nucleic acid conjugates can unlock hybrid systems that outperform either component alone. In...
Sasha B. Ebrahimi, Devleena Samanta· Bioconjugate chemistry· 0 citations
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