차세대 지질나노입자 기반 생체 내 유전체 편집기술 개발
연구책임자 : 이화여자대학교 이혁진 교수
Background
Oligonucleotide therapeutics have emerged as a promising and dynamic class of pharmaceutical agents with remarkable potential for treating a wide spectrum of genetic and acquired diseases. These therapeutic entities, comprising short nucleic acid sequences of either ribonucleic acids (RNA) or deoxyribonucleic acids (DNA), offer the distinct advantage of precise targeting and the ability to interfere with disease-causing genes or proteins. Despite their inherent therapeutic potential, their clinical utility has been hampered by various challenges, including rapid degradation, limited cellular uptake, and unintended immune responses.
Area covered
Chemical modification strategies have been extensively explored to overcome these limitations and enhance their pharmacological properties. In this review, we provide a comprehensive overview of oligonucleotide therapeutics and their associated chemical modification approaches, highlighting their potential in the clinical realm.
Expert opinion
By elucidating the progress made in chemical modifications and their implications for clinical translation, we seek to highlight the pivotal role of these strategies in realizing the full therapeutic potential of oligonucleotide-based therapies for treating a wide range of diseases.
Journal of Pharmaceutical Investigation 2024.02.
