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Galactosemia is a deficiency in the enzymes galactokinase or uridyl transferase, thereby affecting the synthesis and degradation of glycogen synthesis/galactose metabolism. At this point, there is no gene therapy available for treatment - treatment is only through strict dietary control.

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16y ago
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Anonymous

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3y ago

what type of galactosemia is most: sever?

classic (type I) galactokinase deficiency (type II) or: galactose epimerase deficiency (type III) what's wors? please put in order from: worst to not as sever?

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Q: Is there a gene therapy available for galactosemia patients?
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Related questions

Is there reserch into gene therapy for galactosemia?

yes


What is Gene therapy being performed on patients suffering from?

Genetic Disorders


With galactosemia what does the Los Angeles gene refer to?

the city


Is gene therapy available for Wilson's disease?

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What is gene therapy?

Gene therapy is a method of treatment. It is specifically used to treat patients who are suffering from diseases due to defective genes. Essentially, the treatment involves researchers replacing the defective or faulty genes with a normal functioning gene. It involves: 1) Detection of gene 2)Determination & its role 3)Isolation & cloning 4)Introducing the gene by proper way. This is either germline gene therapy (done in germ cells) or somatic gene therapy (done in somatic cells).


Inserting working copies of a gene directly into the cells of a person with a genetic disorder is called what?

Gene TherapyGenetic disorders are caused by an abnormality in person's DNA.


Why do you need gene therapy?

gene therapy is when a faulty gene is repaired or replaced


Difference between gene therapy and protein therapy?

at the gene level gene therapy is done and at the the protein level protein therapy is done


Is Lactose intolerance dominant or recessive trait?

Galactosemia is an autosomal recessive trait.


Biological social ethical economic or environmental implications of gene therapy?

How can "good" and "bad" uses of gene therapy be distinguished?Who decides which traits are normal and which constitute a disability or disorder?Will the high costs of gene therapy make it available only to the wealthy?Could the widespread use of gene therapy make society less accepting of people who are different?Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability?


A technique that involves putting a healthy copy of a gene into cells that have a defective copy of the same gene?

gene therapy.