10/12/2022
🧬Gene Therapy🧬:in the 1980s, advances in molecular biology had already enabled human genes to be sequenced and cloned. Scientists looking for a method of easily producing proteins — such as insulin, the protein deficient in diabetes mellitus type 1 — investigated introducing human genes to bacterial DNA. And in 1990s the US National Healthcare institution approved of the first gene therapy on 4 year old Ashanti de Silva suffering on severe combined immunodeficiency or SCID. The gene therapy worked like this, Ashanti's gene therapy procedure, doctors removed white blood cells from the child's body, let the cells grow in the lab, inserted the missing gene into the cells, and then infused the genetically modified blood cells back into the patient's bloodstream.
What is Gene Therapy: this is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in particular. Gene therapy typically aims to supplement a defective mutant allele with a functional one. Although the technology is still in its infancy. It is used medical procedure as to cure disorders and mutations that would otherwise detrimental.
The basic process of gene therapy is
The gene is inserted into the genome to replace an "abnormal," disease-causing gene. Then A carrier molecule called a vector must be used to deliver the therapeutic gene to the patient's target cells. Currently, the most common vector is a virus that has been genetically altered to carry normal human DNA. Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner and targets specific cells unloading the therapeutic human gene.
Types of gene therapy
In theory gene therapy can change somatic cells or the cells in your body or germline which means like the egg cell and the s***m cell to deliver desired genes and traits to the offspring for somatic it would be divided into two. ex vivo all cells outside the body will be manipulated and in vivo all cells inside the body.
The vectors: 🦠 virus is the most common in usage for gene therapy as they are the perfect instrument due to them inserting genetic materials into the body and hijacking it's function. And producing more copies. The types of vectors are retroviruses,adenoviruses,adeno-associated viruses,naked DNA, envelope protein pseudo typing viral vector.Oligodeoxynucleotides,Lipoplexes and polyplexes.
Broad methods are the normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. This approach is most common.
An abnormal gene could be swapped for a normal gene through homologous recombination.
The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function.
The regulation of what particular gene could be altered.