What is human gene therapy?


Human gene therapy aims to change a gene's expression or the biological characteristics of living cells for therapeutic purposes. Gene therapy is a method for treating or curing disease by changing a person's DNA. Gene treatments can function in a variety of ways:
  • Swapping out a disease-causing gene for a healthy copy
  • Inactivating a disease-causing gene that is not functioning properly
  • The process of adding a new or altered gene to the organism in order to treat a condition
Products utilizing gene therapy are being investigated for the treatment of diseases like cancer, genetic disorders, and infectious diseases. There are a variety of types of gene therapy products, including:
  • Plasmid DNA: The genetic engineering of circular DNA molecules can deliver therapeutic genes to human cells.
  • Viral vectors: Some gene therapy products are generated from viruses because they naturally possess the capacity to introduce genetic material into cells. Viruses that have been altered to make them incapable of spreading infectious disease can then be utilized as vehicles to deliver therapeutic genes into human cells.
  • Bacterial vectors: In order to deliver therapeutic genes into human cells, bacteria can be altered so that they no longer spread infectious diseases.
  • Human gene editing technology: Gene editing aims to remove undesirable genes or fix mutated genes.
  • Patient-derived cellular gene therapy products: The patient's cells are taken out, genetically altered (typically via a viral vector), and then put back in.

The Center for Biologics Evaluation and Research of the FDA is in charge of regulating biological products, including items used in gene therapy (CBER). An investigational new drug application (IND) must be submitted before conducting human clinical research in the US. The submission and approval of a biologics licence application is necessary for the marketing of a gene therapy product (BLA).

Comments

Post a Comment

Popular posts from this blog

What is difference between genetics and genomics?

Image
Genetics is the study of genes and how they affect heredity, or how specific characteristics or disorders are passed down from one generation to the next. The scientific study of genes and their effects is known as genetics. Protein-making instructions, which are carried by genes, control how cells behave and how the body works. Cystic fibrosis, Huntington's disease, and phenylketonuria are a few examples of hereditary or genetic illnesses. Genomics is a more recent term that describes the study of all of a person's genes, including relations of those genes with each other and with the person’s terrain. Genomics includes the scientific study of complex conditions similar as heart complaint, asthma, diabetes, and cancer because these conditions are generally caused more by a combination of inheritable and environmental factors than by individual genes. Genomics is offering new possibilities for curatives and treatments for some complex conditions, as well as new individual sty...
Read more

What diseases can Pre-implantation genetic diagnosis(PGD) detect?

Image
  Pre-implantation genetic diagnosis is a method used in conjunction with in vitro fertilizations, or IVF, that enables testing of embryos for specific traits like their chromosomal make-up as well as testing for genetic illnesses that are handed down via families. In vitro fertilizations  and preimplantation genetic diagnostic tests can be done on a couple's embryos to determine whether one or both partners are carriers of a genetic mutation that could cause a major medical problem in the kid. In the past, couples with a genetic mutation had the option of choosing not to have children or having prenatal testing done with either amniocentesis at around 16 to 18 weeks of pregnancy or with chorionic villus sampling at around 11 to 12 weeks to determine whether the baby was affected by the genetic condition. In case the unborn child has the disease, the couple then has the choice to abort the pregnancy. Nowadays, carriers of genetic illnesses have the option of having IVF and PG...
Read more

What are the difference between Male and Female Genetic?

Image
Men and women share around 20,000 genes in common. Only the physical differences in the sex chromosomes can be distinguished from one another. It is only guys who have the Y chromosome. Despite the X chromosome being present in both sexes, females have two copies while males have just one. The X chromosome is one of two allosomes that determine sex in both men and females of many organisms, including mammals. It is a part of both the XY and the XO sex-determination systems. Early researchers named the X chromosome for its particular characteristics, which led to the subsequent discovery of its counterpart, the Y chromosome , which was named for the following letter in the alphabet. Males have one X and one Y chromosome, while females have two X chromosomes. Early in the development of the female embryo, one of the two X chromosomes is arbitrarily and irreversibly inactivated in cells other than the egg cells. This process is known as X-inactivation or lyonization. The Y chromosome is ...
Read more