Question 1

could gene therapy or transcranial direct current stimulation (tdcs) or any other method help a patient with williams syndrome?

Accepted Answer

Unlikely: There are no gene therapies I know of for this condition.  I have also not seen any research on tDCS use for this condition (mostly depression, parkinson's, gen. anxiety) but very small studies only.  I did review Johns Hopkins Psychiatry and Behavioral Science website as they do some of this research.  It is very limited and no mention of treatment of Williams syndrome.  I am sorry.

Question 2

is cell therapy different from gene therapy?

Accepted Answer

Yes: Cell therapy generally involves stem or pluripotent cells that have the potential, under the right triggers, to grow into many different types of cells. These cells are then transplanted to an area of disease. Gene therapy generally involves replacing small dysfunctional segments of dna. Dna is present in almost every cell in our body and is the instruction manual that tells the cell what to do.

Question 3

what disease is gene therapy is used to treat?

Accepted Answer

Gene therapy: Treating certain congenital enzyme deficiency metabolic disorders. Looks promising in hemophilia where research going. Also lot of interest in cancers and treating infectious diseases.

Question 4

what are the key regulatory issues surrounding gene therapy?

Accepted Answer

Safety: Patient safety is the most important key besides effectiveness.

Question 5

help docs, is gene therapy available with hemochromatosis?

Accepted Answer

No: Do not expect it any time soon. Neglected hemochromatosis will kill you miserably, but it is extremely easy to manage. You may even come to enjoy the phlebotomy treatment. It is saving your life.

Question 6

what is the likelihood for an adult to grow taller with gene therapy?

Accepted Answer

No: No. A person usually stops growing by age 18-24. Gene therapy will have no effect in helping an adult who has finished his growth phase to grow taller.

Question 7

how does gene therapy make me grow taller if my growth plates are already closed?

Accepted Answer

It won't: At 37 years of age, nothing can make you grow taller.

Question 8

what are the genetics tests for cml, can i get gene therapy if they find anything?

Accepted Answer

Chromosome tests: For people with cml, bone marrow chromosome studies (karyotype) often show the philadelphia chromosome (a chromosome #22 that is shortened because its distal end is transferred (translocated) to chromosome 9. Antibody (gleevec) to the translocation protein is helpful and bone marrow transplant is a form of gene therapy that provides cells with normal chromsomes.

Question 9

how should gene therapy treat certain diseases?

Accepted Answer

By replacing defect: Gene therapy is the use of genes for therapeutic effect.  The original studies with gene therapy were for corrective gene therapy.  To illustrate, patients with severe combined immunodefeciency were born with a mutation in the ada gene.  The original gene therapy trial, engineered a virus that was used to infect bone marrow of children with this disorder.  The normal gene corrected the disorder.

Question 10

is the gene therapy for aids?

Accepted Answer

Gene therapy for HIV: Gene therapy for HIV could be coming. A company called sangamo pharm. Has developed a gene modification tool called a zinc-finger endonuclease that can alter on of the lymphocyte genes that allows HIV to enter the cells; called a chemokine receptor. They are involved in clinical studies at this time that look promising.

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Doctor answers about:

Genes and gene therapy williams syndrome

A member asked:

Could gene therapy or transcranial direct current stimulation (tdcs) or any other method help a patient with williams syndrome?

A member asked:

Is cell therapy different from gene therapy?

A member asked:

What disease is gene therapy is used to treat?

A member asked:

What are the key regulatory issues surrounding gene therapy?

A member asked:

Help docs, is gene therapy available with hemochromatosis?

A member asked:

What is the likelihood for an adult to grow taller with gene therapy?

A member asked:

How does gene therapy make me grow taller if my growth plates are already closed?

A member asked:

What are the genetics tests for cml, can i get gene therapy if they find anything?

A member asked:

How should gene therapy treat certain diseases?

A member asked:

Is the gene therapy for aids?

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