Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological Diseases

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 i...

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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
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container_title Nanomaterials (Basel, Switzerland)
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creator 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
description 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.
doi_str_mv 10.3390/nano13101680
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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
title Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological Diseases
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