Chapter 1 : Introduction
To The Gene and Gene Therapy
What is a gene
?
Genes basically are
small molecules that carry our genetic information. Genes consist of many nucleotides
that make up our DNA, and are normally found in thread like structure called Chromosomes.
Those chromosomes are usually within the nucleus of each human cell. When the
cell divide and produce new daughter cells, a complete set of chromosomes can
be found in each daughter cell. Inside each cell of our body, there are two copies
of each gene; one from each parent. The main function of our genes is that it gives
the instructions of synthesizing proteins
and enzymes, which are important for the body functions and metabolisms. Sometimes,
abnormal genes arises from mutations, which are alteration of the nucleotide
sequence. As a result, the gene produce missing or un-functional proteins that
may cause genetic disorders.
What is Gene Therapy ?
Gene Therapy can
correct the genetic defects that mutations may resulted by targeting the
abnormal gene and inserting new one to the cell. If mutations in certain gene
resulted in abnormal or missing proteins, then the protein will not be able to
function normally. Gene therapy can
introduce a normal copy of the gene to make the protein functional. The first successful
gene therapy was done by William French Anderson in 1990 at the National
Institutes of Health (NIH). He was successfully able to treat a girl who was
suffering from Severe Combined Immunodeficiency disease that is known of
producing impaired immune function. Nowadays,
there are many patients receiving gene therapy correcting their faulty proteins
and making them function normally. However, gene therapy is a very challenging task. For instance, scientists have to find ways to transfer
a foreign gene to the human call, make the gene expressed, and finally avoid
the immune response to this foreign molecule.
References :
http://ghr.nlm.nih.gov/handbook/therapy
Pierce,
B. A. (2012). Genetics a conceptual approach. New York, NY: Kate Ahr Parker.
Hi Zainab!
ReplyDeleteGreat first post. Short, sweet, and easy to read and understand. A good overview of both the structure and function of a gene. A short background of gene therapy was nice. Seems like a daunting task to me! Hopefully scientists can overcome the challenges you have listed here so we can make effective use of this as a therapy.
Great overview of why genes are important, especially using language that non-scientists can handle. I wonder if maybe an example or two of a disease or condition that people recognize that's treated with gene therapy might help some have an idea about how common gene therapy is, but I'm curious to read more.
ReplyDelete