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Gene therapy

2010-11-24T15:13:30Z

090147375:

Although still a very experimental approach, [[Gene_therapy|Gene Therapy]] techniques are currently being developed to treat and prevent many diseases such as [[Cystic fibrosis|Cystic Fibrosis]]. This includes many diseases that are as yet incurable, such as cancers and many genetically inherited diseases.

Current approaches being investigated are:

1. The "knock out" gene approach - Inactivation or removal of genes that have been mutated and/or do not function in the desired way.

2. The "Replacement" approach - Replacement of disease causing gene mutations with a healthy copy of said gene.

3. The new gene approach - Introduction of a completely new gene into the patient.

[[Gene_therapy|Gene Therapy]] techiniques still remain extremely risky and are still very much in the early stages of development, little is known about the side effects of gene manipulation in human patients.<ref>Genetics Home Reference,What is gene therapy?, published 22/11/10, Last viewed 24/11/10fckLRhttp://ghr.nlm.nih.gov/handbook/therapy/genetherapy</ref>

=== References ===

<references />

Gene therapy

2010-11-24T14:41:56Z

090147375:

Although still a very experimental approach, Gene Therapy techniques are currently being developed to treat and prevent many diseases such as Cystic Fibrosis. This includes many diseases that are as yet incurable, such as cancers and many genetically inherited diseases.

Current approaches being investigated are:

1. The "knock out" gene approach - Inactivation or removal of genes that have been mutated and/or do not function in the desired way.

2. The "Replacement" approach - Replacement of disease causing gene mutations with a healthy copy of said gene.

3. The new gene approach - Introduction of a completely new gene into the patient.

Gene Therapy techiniques still remain extremely risky and are still very much in the early stages of development, little is known about the side effects of gene manipulation in human patients.<references /><ref>http://ghr.nlm.nih.gov/handbook/therapy/genetherapy</ref>

Gene therapy

2010-11-24T14:41:29Z

090147375: Created page with 'Although still a very experimental approach, Gene Therapy techniques are currently being developed to treat and prevent many diseases such as Cystic Fibrosis. This includes many …'

Although still a very experimental approach, Gene Therapy techniques are currently being developed to treat and prevent many diseases such as Cystic Fibrosis. This includes many diseases that are as yet incurable, such as cancers and many genetically inherited diseases.

Current approaches being investigated are:

1. The "knock out" gene approach - Inactivation or removal of genes that have been mutated and/or do not function in the desired way.

2. The "Replacement" approach - Replacement of disease causing gene mutations with a healthy copy of said gene.

3. The new gene approach - Introduction of a completely new gene into the patient.

Gene Therapy techiniques still remain extremely risky and are still very much in the early stages of development, little is known about the side effects of gene manipulation in human patients.<ref>http://ghr.nlm.nih.gov/handbook/therapy/genetherapy</ref>

CFTR

2010-11-11T11:31:01Z

090147375:

CFTR is the name commonly given to the gene responsible for the production of the [[Cystic fibrosis|Cystic Fibrosis]] Transmembrane Regulator [[Proteins|protein]]. This gene, when mutated, can cause [[Cystic fibrosis|cystic fibrosis]], although it is [[Recessive gene|recessive]], whether the mutations are identical or not. So far, over 1500 mutations of the [[CFTR|CFTR]] gene have been discovered, the most common being the ∆F508 mutation, sometimes in as few as one individual.

CFTR is a member of the [[ABC_Superfamily|ABC superfamily ]]of proteins and is the only Cl<sup>-</sup> channel in this family. It is very similar to [[P-glycoprotein|P-glycoprotein]]. The CFTR gene was cloned in 1989.

CFTR is a passive transporter and it is special in the [[ABC_Superfamily|ABC superfamily]] proteins. It has got 12 transmembrane domains, with two nucleotide binding domains (NBD1 and NBD2) and a regulatory domain. The binding of [[ATP|ATP is]] essential for the function for the channel, NBD1 and NBD2 both bind [[ATP|ATP however]] only NBD2 can hydrolyse [[ATP|ATP]].