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Related Concept Videos

Gene Therapy00:59

Gene Therapy

25.6K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Methods of Nuclear Reprogramming01:24

Methods of Nuclear Reprogramming

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Nuclear reprogramming is a process of transforming one cell type into an unrelated cell type by epigenetic changes that alter the cell’s original gene expression pattern. Such epigenetic changes force cells to express a different set of genes, which play a significant role in inducing transformation into other cell types. Nuclear reprogramming offers applications in reproductive cloning for livestock propagation and regenerative medicine — developing patient-specific cells for...
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Introduction to Nuclear Reprogramming01:14

Introduction to Nuclear Reprogramming

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Nuclear reprogramming is the process of switching gene expression of one cell type to that of another cell type, usually from a differentiated cell state to an undifferentiated cell state. Differentiation occurs during processes such as development and morphogenesis, tissue regeneration, and malignancy. Cells can also be artificially induced to reprogram their gene expression by techniques such as nuclear transfer, induced pluripotency, and cell fusion. Such techniques have many applications in...
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Related Experiment Video

Updated: Jul 29, 2025

Lineage-reprogramming of Pericyte-derived Cells of the Adult Human Brain into Induced Neurons
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Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological Diseases.

Yujung Chang1,2, Sungwoo Lee3, Jieun Kim4

  • 1Laboratory of Regenerative Medicine for Neurodegenerative Disease, Stand Up Therapeutics, Hannamdaero 98, Seoul 04418, Republic of Korea.

Nanomaterials (Basel, Switzerland)
|May 27, 2023
PubMed
Summary

Gene therapy shows promise for neurological disorders like Parkinson's disease. This study explores nanoporous particles to enhance direct lineage reprogramming efficiency for treating these conditions.

Keywords:
cell fate conversiondirect lineage reprogramminggene therapynanoporous particle-based gene deliveryspinal cord injury

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Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases
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Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases

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Area of Science:

  • Regenerative Medicine
  • Neuroscience
  • Biotechnology

Background:

  • Gene therapy offers a novel approach to treating neurological diseases by transferring genetic material into patient cells.
  • Adeno-associated viruses are being investigated for targeted gene delivery in neurological conditions such as Parkinson's disease and spinal cord injury.
  • Direct lineage reprogramming (DLR) presents an alternative to stem cell therapy for incurable diseases, but faces efficiency challenges.

Purpose of the Study:

  • To investigate innovative strategies for enhancing the efficiency of direct lineage reprogramming (DLR) for neurological applications.
  • To explore the use of nanoporous particle-based gene delivery systems to improve DLR-induced neuron generation.
  • To advance the development of effective gene therapies for debilitating neurological disorders.

Main Methods:

  • Utilized a nanoporous particle-based gene delivery system.
  • Focused on improving the reprogramming efficiency of direct lineage reprogramming (DLR).
  • Investigated strategies to overcome limitations in clinical DLR application.

Main Results:

  • The study focused on innovative strategies to improve DLR efficiency.
  • Nanoporous particle-based gene delivery was explored as a method to enhance reprogramming.
  • The research aims to facilitate the development of more effective gene therapies.

Conclusions:

  • Innovative strategies, including nanoporous particle-based gene delivery, are crucial for enhancing DLR efficiency.
  • Improving DLR efficiency is key to its clinical application for neurological disorders.
  • This research contributes to the advancement of gene therapy for conditions like Parkinson's disease and spinal cord injury.