Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological Diseases
Yujung Chang,
Sungwoo Lee,
Jieun Kim,
Chunggoo Kim,
Hyun Soo Shim,
Seung Eun Lee,
Hyeok Ju Park,
Jeongwon Kim,
Soohyun Lee,
Yong Kyu Lee,
Sungho Park,
Junsang Yoo
Affiliations
Yujung Chang
Laboratory of Regenerative Medicine for Neurodegenerative Disease, Stand Up Therapeutics, Hannamdaero 98, Seoul 04418, Republic of Korea
Sungwoo Lee
Department of Chemistry, Sungkyunkwan University, 2066, Seobu-ro, Jangan-gu, Suwon-si 16419, Republic of Korea
Jieun Kim
Department of Bio-Health Technology, College of Biomedical Science, Kangwon National University, 1 Kangwondeahak-gil, Chuncheon 24341, Republic of Korea
Chunggoo Kim
Laboratory of Regenerative Medicine for Neurodegenerative Disease, Stand Up Therapeutics, Hannamdaero 98, Seoul 04418, Republic of Korea
Hyun Soo Shim
Laboratory of Regenerative Medicine for Neurodegenerative Disease, Stand Up Therapeutics, Hannamdaero 98, Seoul 04418, Republic of Korea
Seung Eun Lee
Research Animal Resource Center, Korea Institute of Science and Technology, Hwarang-ro 14-gil, Seongbuk-gu, Seoul 02792, Republic of Korea
Hyeok Ju Park
Database Laboratory, Department of Computer Science and Engineering, Dongguk University-Seoul, Pildong-ro 1-gil 30, Jung-gu, Seoul 04620, Republic of Korea
Jeongwon Kim
Department of Chemistry, Sungkyunkwan University, 2066, Seobu-ro, Jangan-gu, Suwon-si 16419, Republic of Korea
Soohyun Lee
Department of Chemistry, Sungkyunkwan University, 2066, Seobu-ro, Jangan-gu, Suwon-si 16419, Republic of Korea
Yong Kyu Lee
Database Laboratory, Department of Computer Science and Engineering, Dongguk University-Seoul, Pildong-ro 1-gil 30, Jung-gu, Seoul 04620, Republic of Korea
Sungho Park
Department of Chemistry, Sungkyunkwan University, 2066, Seobu-ro, Jangan-gu, Suwon-si 16419, Republic of Korea
Junsang Yoo
Laboratory of Regenerative Medicine for Neurodegenerative Disease, Stand Up Therapeutics, Hannamdaero 98, Seoul 04418, Republic of Korea
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.
