Clinical Therapeutics, Год журнала: 2024, Номер unknown
Опубликована: Окт. 1, 2024
Язык: Английский
Journal of Nanotheranostics, Год журнала: 2025, Номер 6(2), С. 10 - 10
Опубликована: Апрель 9, 2025
Nanotheranostics—where nanoscale materials serve both diagnostic and therapeutic functions—are rapidly transforming gene therapy by tackling critical delivery challenges. This review explores the design engineering of various nanoparticle systems (lipid-based, polymeric, inorganic, hybrid) to enhance stability, targeting, endosomal escape genetic payloads. We discuss how real-time imaging capabilities integrated into these platforms enable precise localization controlled release genes, improving treatment efficacy while reducing off-target effects. Key strategies overcome barriers (such as proton sponge effect photothermal disruption) achieve nuclear are highlighted, along with recent advances in stimuli-responsive that facilitate spatiotemporal control expression. Clinical trials preclinical studies demonstrate expanding role nanotheranostics managing cancer, inherited disorders, cardiovascular neurological diseases. further address regulatory manufacturing hurdles must be for widespread clinical adoption nanoparticle-based therapies. By synthesizing progress ongoing challenges, this underscores transformative potential effective, targeted, image-guided delivery.
Язык: Английский
Процитировано
2
Cells, Год журнала: 2024, Номер 13(11), С. 920 - 920
Опубликована: Май 27, 2024
Diamond–Blackfan anemia (DBA) is a rare genetic disorder affecting the bone marrow’s ability to produce red blood cells, leading severe and various physical abnormalities. Approximately 75% of DBA cases involve heterozygous mutations in ribosomal protein (RP) genes, classifying it as ribosomopathy, with RPS19 being most frequently mutated gene. Non-RP mutations, such GATA1, have also been identified. Current treatments include glucocorticosteroids, transfusions, hematopoietic stem cell transplantation (HSCT), HSCT only curative option, albeit challenges like donor availability immunological complications. Gene therapy, particularly using lentiviral vectors CRISPR/Cas9 technology, emerges promising alternative. This review explores potential gene focusing on technology combination non-integrating vectors, solution for DBA. It highlights transformative advancements treatment landscape DBA, offering hope individuals affected by this condition.
Язык: Английский
Процитировано
2
Immunology, Год журнала: 2024, Номер unknown
Опубликована: Дек. 16, 2024
ABSTRACT The story of nucleases begins on the ancient battlefields early Earth, where simple bacteria fought to survive against viral invaders. Nucleases are enzymes that degrade nucleic acids, with restriction endonucleases emerging as some earliest defenders, cutting foreign DNA protect their hosts. However, sought more than just defence. They evolved CRISPR‐Cas system, an adaptive immune mechanism capable remembering past now‐famous Cas9 nuclease, a key player in this has been harnessed for genome editing, revolutionising biotechnology. Over time, from basic defence tools into complex regulators function higher organisms. In humans, DNases and RNases maintain balance by clearing cellular debris, preventing autoimmunity, defending pathogens. These have transformed bacterial defenders critical players both human immunity This review explores evolutionary history vital roles protectors life's mechanisms.
Язык: Английский
Процитировано
0
Clinical Therapeutics, Год журнала: 2024, Номер unknown
Опубликована: Окт. 1, 2024
Язык: Английский
Процитировано
0