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Related Concept Videos

Gene Therapy00:59

Gene Therapy

25.4K
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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Overview
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The Central Dogma01:20

The Central Dogma

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The central dogma explains the flow of genetic information from DNA nucleotides to the amino acid sequence of proteins.
RNA is the Missing Link Between DNA and Proteins
In the early 1900s, scientists discovered that DNA stores all the information needed for cellular functions and that proteins perform most of these functions. However, the mechanisms of converting genetic information into functional proteins remained unknown for many years. Initially, it was believed that a single gene is...
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iPS Cell Differentiation01:22

iPS Cell Differentiation

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The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
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In-vitro Mutagenesis01:16

In-vitro Mutagenesis

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To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
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CRISPR01:59

CRISPR

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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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Related Experiment Video

Updated: Jul 2, 2025

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
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Recent developments in gene therapy research in India.

Ruchita Selot1, Arkasubhra Ghosh

  • 1GROW Research Laboratory, Narayana Nethralaya Foundation, Bengaluru 560099, India.

Journal of Biosciences
|February 22, 2024
PubMed
Summary
This summary is machine-generated.

Gene therapy offers a promising treatment for inherited genetic disorders, with viral vectors like adeno-associated virus (AAV) showing significant potential. India is advancing its gene therapy research and clinical trials, addressing challenges to benefit its large rare disease population.

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Inherited genetic disorders are progressive, often leading to severe organ dysfunction or death, with over 95% lacking permanent treatment options.
  • Gene therapy, involving gene restoration, replacement, inhibition, or editing, presents a promising molecular approach for treating rare genetic diseases affecting millions globally.
  • Viral vectors, particularly adeno-associated virus (AAV) and lentivirus, are increasingly utilized in gene therapy clinical trials due to their efficiency.

Purpose of the Study:

  • To outline gene therapy research, regulatory processes, and clinical trial launches in India.
  • To contextualize India's gene therapy advancements within global developments.
  • To highlight ongoing research, product manufacturing, and community initiatives in India's gene therapy landscape.

Main Methods:

  • Review of current gene therapy research and clinical trial data, focusing on viral vector applications.
  • Analysis of factors influencing gene therapy development and adoption in India, including infrastructure and investment.
  • Discussion of regulatory frameworks and guidelines for gene therapy clinical trials in India.

Main Results:

  • Adeno-associated virus (AAV)-mediated gene therapy has demonstrated high potential in clinical trials for various conditions.
  • Significant financial investment, technological development, and trained manpower are crucial for gene therapy product manufacturing and approval.
  • India is increasing its gene therapy development, spurred by global approvals and growing rare disease awareness, despite historical infrastructure challenges.

Conclusions:

  • Gene therapy holds immense promise for treating rare genetic disorders in India's large patient population.
  • Addressing challenges in infrastructure, patient data, and investment is key to advancing gene therapy in India.
  • Collaborative efforts between the medical community, patient groups, and regulatory agencies are vital for successful gene therapy implementation.