Chang, Yujung Lee, Sungwoo Kim, Jieun Kim, Chunggoo Shim, Hyun Soo Lee, Seung Eun Park, Hyeok Ju Kim, Jeongwon Lee, Soohyun Lee, Yong Kyu Park, Sungho Yoo, Junsang 2024-01-19T09:32:51Z 2024-01-19T09:32:51Z 2023-06-15 2023-05 2079-4991 https://pubs.kist.re.kr/handle/201004/113748 Gene therapy is an innovative approach in the field of regenerative medicine. This therapy entails the transfer of genetic material into a patient's cells to treat diseases. In particular, gene therapy for neurological diseases has recently achieved significant progress, with numerous studies investigating the use of adeno-associated viruses for the targeted delivery of therapeutic genetic fragments. This approach has potential applications for treating incurable diseases, including paralysis and motor impairment caused by spinal cord injury and Parkinson's disease, and it is characterized by dopaminergic neuron degeneration. Recently, several studies have explored the potential of direct lineage reprogramming (DLR) for treating incurable diseases, and highlighted the advantages of DLR over conventional stem cell therapy. However, application of DLR technology in clinical practice is hindered by its low efficiency compared with cell therapy using stem cell differentiation. To overcome this limitation, researchers have explored various strategies such as the efficiency of DLR. In this study, we focused on innovative strategies, including the use of a nanoporous particle-based gene delivery system to improve the reprogramming efficiency of DLR-induced neurons. We believe that discussing these approaches can facilitate the development of more effective gene therapies for neurological disorders. English MDPI Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological Diseases Article 10.3390/nano13101680 1 Nanomaterials, v.13, no.10 Nanomaterials 13 10 Y scie scopus 000996702700001 2-s2.0-85160435896 Chemistry, Multidisciplinary Nanoscience & Nanotechnology Materials Science, Multidisciplinary Physics, Applied Chemistry Science & Technology - Other Topics Materials Science Physics Review LEBER CONGENITAL AMAUROSIS BLOOD-BRAIN-BARRIER DIRECT CONVERSION IN-VIVO PARKINSONS-DISEASE DOPAMINE NEURONS FUNCTIONAL-NEURONS NONHUMAN-PRIMATES HUMAN FIBROBLASTS OPEN-LABEL cell fate conversion direct lineage reprogramming spinal cord injury gene therapy nanoporous particle-based gene delivery