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Is gene therapy future of medical treatment?

What are genes?

Genes are located n the nucleus of human cells, as part of structures called chromosomes. They are composed of DNA and proteins. A small proportion of genes are located outside the nucleus, in the mitochondria.

What is the location of 2 genes in human cells?

The majority of genes are found in the DNA of the nucleus, which contains the cell’s complete genome. Each chromosome contains thousands of genes organised along the DNA strand. The DNA is tightly packed and coiled around proteins called histones

What is genomics?

Genomics is the study of the structure, function, evolution and mapping of an organism’s entire genome, the complete set of its DNA, including all its genes. Genetics focuses on individual genes, genomics examines all of the genes and their interactions within an organism and with the environment.

 

Any challenges and limitations?

The therapeutic gene should reach the right cells without causing any harm. The body may reject the viruses that are the transporters of these genes. Currently the therapy is extremely expensive. There could be unintended genetic changes and they can cause new diseases. They could affect future generations if reproductive genes are intervened. This may lead to ethical concerns as it may affect future generations.

Does genomics have any applications?

It can identify genetic mutations causing diseases. Say in sickle cell disease or BRCA 1/BRCA2 for breast cancer. It can also design treatment as per the individual’s genetic profile. Some blood thinners and anti cancer drugs and HIV drugs may come under this category. Gene therapy can treat certain genetic disorders. It can also optimise drug therapy based on genetic makeup and can reduce adverse drug reactions.

Status of gene therapy in India?

This is an emerging field with significant potential to transform health care. Though still in its early stages, the key targets for gene therapy in India include beta-thalassemia , sickle cell anaemia, spinal muscular atrophy. A global CRISPR based therapy is being evaluated. CAR-T cell therapy to target cancer cells has been introduced at research centres.

 

Which institutions in India are currently conducting research in gene therapy?

Centre for Cellular and Molecular Biology Hyderabad is leading research in genetic disorders. Indian Institute of Science, Bengaluru is working on CRISPR based gene editing, National Institute of Immunology New Delhi is exploring gene based treatments for various diseases. Tata Memorial Hospital, Mumbai is conducting CAR-T therapy trials for blood cancers.

What are the advantages of gene therapy?

Unlike drugs that manage symptoms, gene therapy addresses the disease at its genetic source. A single treatment course may provide long lasting or permanent cure. Treatment effectiveness is vastly improved. It eliminates the need for lifelong medications or repeated treatments for chronic conditions.

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