Gene Therapy


Difference between Gene therapy and Gene editing:

All concepts of Gene therapy, Gene editing and CRISPR CAS9 are interlinked. We use Gene editing for multiple reasons like designer babies, treatment of genetic disorders, for invention of medicines etc., if we are editing Gene for health related then its called Gene therapy. Besides there is also difference of degree, in Gene therapy we don’t replace the  Gene

In gene editing, a mutated gene is revised, removed, or replaced at the DNA level. In gene therapy, the effect of a mutation is offset by inserting a “healthy” version of the gene, and the disease-related genes remain in the genome. Both approaches may provide a durable benefit to patients, and both gene therapy and gene editing, alone or in combination, may lend themselves to the development of transformative genomic medicines.


Genes, Proteins, and DNA

The human body is made up of trillions of cells.Each cell has a copy of your genome—the total collection of all your genes and DNA.Genes are instructions that every cell in your body uses to make the proteins they need to function.Genes can be found in long chains of molecules called deoxyribonucleic acids (DNA) that are twisted together into the shape of a double helix.

There are 4 DNA molecules that are identified by the letters A, T, C, and G. Combinations of these letters make up the genetic instructions that our cells use to make proteins.Our genes can also be a source of disease.

Small breaks in our DNA are incredibly common and are normally uneventful. DNA breaks can happen from sunlight, for example, or during cell divisions that happen as we grow. Our cells have built-in DNA repair processes that constantly fix these breaks as they occur.

However, breaks are sometimes repaired incorrectly, creating what is known as a mutation. Mutations can occur spontaneously or be passed down from our parents. Mutations can also change how our cells function, and may lead to serious diseases such as sickle cell disease (SCD), Leber congenital amaurosis 10 (LCA10), cancer, and many others.


Gene therapy is a technique that modifies a person’s genes to treat or cure disease. Gene therapies can work by several mechanisms:

1.Replacing a disease-causing gene with a healthy copy of the gene

2.Inactivating a disease-causing gene that is not functioning properly

3.Introducing a new or modified gene into the body to help treat a disease

Gene therapy products are being studied to treat diseases including cancer, genetic diseases, and infectious diseases.



1. Somatic Gene Therapy: Effects will not be transferred to next generation

2.Germline Gene Therapy: Effects transferred to next generation



Response from immune system: our immune system might not accept newly introduced gene

Mutation of undesired Gene and collateral effect

High cost for treatment of disease through Gene therapy

Delivery of desired Gene to right place

Scientists can make mistakes and it can lead to occurrence of unwanted mutations

Possibility of Bio war

Virus is used as vector and it can show toxicity