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Related Concept Videos

Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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Neurogenesis and Regeneration of Nervous Tissue01:15

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In the CNS, neurogenesis, the birth of new neurons from stem cells, is limited to the hippocampus in adults. In other regions of the brain and spinal cord, neurogenesis is almost non-existent due to inhibitory influences from neuroglia, especially oligodendrocytes, and the absence of growth-stimulating cues. The myelin produced by oligodendrocytes in the CNS inhibits neuronal regeneration. Furthermore, astrocytes proliferate rapidly after neuronal damage, forming scar tissue that physically...
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Gene Therapy00:59

Gene Therapy

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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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Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases
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Genetic therapy for the nervous system.

William J Bowers1, Xandra O Breakefield, Miguel Sena-Esteves

  • 1Department of Neurology, Center for Neural Development and Disease, University of Rochester, School of Medicine and Dentistry, Rochester, NY 14642, USA.

Human Molecular Genetics
|March 25, 2011
PubMed
Summary
This summary is machine-generated.

Genetic therapy is advancing rapidly, offering promising new treatments for neurological disorders. This review explores various genetic strategies and technologies for ameliorating conditions like Alzheimer

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Area of Science:

  • Neuroscience and Genetics
  • Molecular Biology
  • Biotechnology

Background:

  • Genetic therapy is experiencing a significant resurgence, driven by advancements in vector technology and molecular tools.
  • Neurological syndromes represent a diverse group of debilitating conditions with unmet therapeutic needs.
  • Existing treatments for many neurological diseases are limited, highlighting the need for innovative approaches.

Purpose of the Study:

  • To review current and emerging genetic strategies for treating a wide range of neurological syndromes.
  • To highlight the role of novel genetic technologies in developing targeted therapies.
  • To discuss the potential of gene therapy in clinical trials for neurological disorders.

Main Methods:

  • Review of scientific literature on genetic therapies for neurological diseases.
  • Analysis of advancements in viral and synthetic vectors, oligonucleotides, and genetically modified cells.
  • Examination of genetic technologies for gene identification, transcriptional regulation, and RNA-based interventions.

Main Results:

  • Genetic strategies show promise for ameliorating various neurological syndromes, including lysosomal storage diseases, Alzheimer's disease, Parkinson's disease, spinal muscular atrophy, amyotrophic lateral sclerosis, and brain tumors.
  • Clinical trials utilizing these genetic approaches are yielding encouraging results.
  • New tools enable precise manipulation of genetic elements for tailored therapeutic interventions.

Conclusions:

  • Genetic therapy offers a powerful and versatile approach to treating complex neurological disorders.
  • Continued innovation in genetic technologies is crucial for advancing the field and improving patient outcomes.
  • The personalized nature of some genetic interventions, such as using patient-derived induced pluripotent stem cells, holds significant therapeutic potential.