Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological Diseases

• Published in: Nanomaterials (Basel, Switzerland) Switzerland), 2023-05, Vol.13 (10), p.1680
• Main Authors: 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
• Format: Article
• Language: English
• Subjects: Alzheimer's disease; Biomedical engineering; Brain research; Cell differentiation; cell fate conversion; Cell therapy; Degeneration; Differentiation (biology); direct lineage reprogramming; Dopamine; Dopamine receptors; Efficiency; Fibroblasts; Gene therapy; Gene transfer; Genes; Genetic engineering; Health aspects; Kinases; Li Siguang; Movement disorders; Nanoparticles; nanoporous particle-based gene delivery; Nanotechnology; Nervous system diseases; Neurodegeneration; Neurodegenerative diseases; Neurological diseases; Neurons; Oxidative stress; Paralysis; Parkinson's disease; Regenerative medicine; Review; Spinal cord; Spinal cord injuries; spinal cord injury; Stem cell research; Stem cells; Transcription factors; Transplantation
• ISSN: 2079-4991; 2079-4991
• DOI: 10.3390/nano13101680

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.