Gene Therapy: A Revolutionary Strategy to Genetic Disorder Treatment

Gene therapy is currently an important subject in the biotech sector, with numerous medicines under research and various recent approvals. However, the way has not always been easy. It has been one of the biggest achievements of the twenty-first century. Genetic disorders were formerly thought to be incurable, engraved in stone within the genomes of individuals unfortunate enough to be born with them in genetic life. In this blog, let’s explore gene therapy.

A growing number of firms are entering the market. US FDA expects to receive more than 200 new applications for cell and gene therapy each year, with 10-20 therapies approved each year. Thus, this factor is anticipated to boost the market growth. In addition, according to a research report by Astute Analytica, the Global Gene Therapy Market is likely to increase at a compound annual growth rate (CAGR) of 24% over the forecast period from 2023 to 2030.

Historical Overview of Gene Therapy:

In 1972, US scientists Richard Roblin and Theodore Friedmann released a study in Science titled ‘Gene therapy for human genetic disease?’ in which they discussed the enormous potential of inserting DNA sequences into patients’ cells for treating people with genetic illnesses. They did, however, urge caution in the technology’s growth, pointing out numerous important barriers to scientific knowledge that needed to be overcome.

Following 18 years of more research, the first gene therapy experiment began in 1990. A four-year-old girl called Ashanthi DeSilva had a 12-day treatment for severe combined immunodeficiency, a rare genetic illness. DeSilva lacked a critical enzyme known as adenosine deaminase (ADA), which damaged her immune system and put her at risk of developing a potentially fatal infection.

Gene Therapy Products: Gene therapy products are being researched for the treatment of diseases such as cancer, hereditary diseases, and viral infections.

Wide range of gene therapy products, such as:

Viral vectors: Viruses can naturally deliver genetic material into cells, and several gene therapy products are developed from viruses. Once viruses have been changed so that they no longer have the ability to cause infectious disease, they can be employed as vectors (vehicles) to deliver therapeutic genes into human cells.

Human gene editing technological advances: Gene editing aims to either disrupt dangerous genes or fix mutated genes.

Bacterial vectors: Bacteria can be created to avoid producing infectious illnesses and then utilized as vectors (vehicles) to deliver therapeutic genes into human cells.

Products generated from patients for cellular gene therapy: Cells are extracted from the patient, physically changed (sometimes with the help of a viral vector), and back to the patient.

Plasmid DNA: Therapeutic genes can be delivered into human cells via circular DNA molecules that have been genetically modified.

The Current Situation Of Gene Therapy Several gene therapies have been approved by regulatory organizations such as the FDA for the treatment of several ailments such as uncommon diseases, certain types of cancer, genetic disorders, and inherited eye diseases. However, numerous problems remain in the research and administration of gene therapies, such as safety concerns, ethical concerns, and the high price of treatment. Source: A Revolutionary Strategy to Genetic Disorder Treatment