Related Experiment Video
Updated: Sep 19, 2025
03:52
Author Spotlight: Efficient Adeno-Associated Virus Isolation for Pre-Clinical Applications
Published on: February 9, 2024
3.0K
Current perspectives on gene therapy and its involvement in curing genetic disorders
Shireen Nishad1, Dipali Dongare1, Sayani Saha1
1Department of Biotechnology, National Institute of Pharmaceutical Education and Research, Bijnor-Sisendi Road, Sarojini Nagar, Near CRPF Base Camp, Lucknow, UP, 226002, India.
Human Genetics
|June 18, 2025
Summary
Genomics advances enable personalized medicine by identifying genetic risks for diseases like cancer. Gene-editing tools like CRISPR offer precise treatments for genetic disorders, improving public health outcomes.
Area of Science:
- Genomic Medicine
- Medical Science
Background:
- Genomics is transforming medicine through whole-genome sequencing.
- The Human Genome Project identified genetic variations linked to diseases, enabling personalized medicine.
- Genomic research targets rare genetic disorders and enhances public health.
Purpose of the Study:
- To review the impact of genomics on public health.
- To highlight the potential of genomics in reshaping healthcare.
- To discuss innovative treatment strategies enabled by genomics.
Main Methods:
- Review of advances in whole-genome sequencing.
- Analysis of the Human Genome Project's contributions.
- Examination of gene-editing technologies like CRISPR.
Main Results:
- Genomics enables genome-based diagnostics and personalized therapies.
- Identification of genetic variations associated with increased disease risks (e.g., cancer).
- Development of precise therapies for rare genetic disorders (e.g., Spinal muscular atrophy, Parkinson's disease).
Conclusions:
- Genomics is foundational to personalized medicine.
- Gene-editing tools like CRISPR improve treatment efficacy with minimal side effects.
- Genomics holds significant potential to reshape healthcare and enhance public health.
More Related Videos
Related Concept Videos
00:59Gene Therapy
25.9K
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...
25.9K
00:49What is Genetic Engineering?
75.6K
Overview
75.6K
01:29Microorganisms in Medicine and Therapeutics
375
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.
375
01:59CRISPR
53.0K
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...
53.0K
01:28Human Genetics
741
Human genetics provides a profound framework for understanding the interplay between genetic predispositions and human psychology. At the heart of this discipline lies the study of how genes influence physical traits, behaviors, and susceptibility to diseases. Each person carries a unique genetic code that subtly or significantly shapes their psychological and behavioral landscape.
The complex relationship between genetics and psychology is observable through common biological components such...
The complex relationship between genetics and psychology is observable through common biological components such...
741
01:20The Central Dogma
28.5K
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...
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...
28.5K