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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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17y ago

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Is there reserch into gene therapy for galactosemia?

yes


What is Gene therapy being performed on patients suffering from?

Genetic Disorders


In the SCID-X1 gene therapy trials what problem later surfaced in three of the patients?

In the SCID-X1 gene therapy trials, three patients developed leukemia as a result of the treatment. This was due to the unexpected activation of an oncogene during the insertion of the corrective gene into the patients' cells.


Is gene therapy available for Wilson's disease?

yes


With galactosemia what does the Los Angeles gene refer to?

the city


Where does one go to get gene therapy?

Gene therapy is an experimental treatment in which genetic material is introduced into a person's cells to cure disease. Because it is still experimental it is being done at research hospitals.


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?

Lactose intolerance is typically considered a recessive trait. It is caused by a reduced ability to produce the enzyme lactase, which digest lactose. If an individual inherits two copies of the gene associated with lactose intolerance (one from each parent), they are likely to be lactose intolerant.


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?

That process is called gene therapy. It involves inserting functional copies of a gene into the cells of a person with a genetic disorder to correct the genetic mutation causing the disorder.


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?


How might informed consent be a problem with gene therapy?

Informed consent for gene therapy can be challenging due to the complexity of the technology and potential long-term consequences. Patients may have difficulty understanding the risks and benefits, leading to incomplete or inaccurate consent. Additionally, individuals may feel pressured to participate in gene therapy trials without fully comprehending the implications.