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Doctor insights on: Genes And Gene Therapy Williams Syndrome

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Genetic (Definition)

From a medical standpoint, "genetic" refers to the potential heritability of various medical conditions. While some conditions are inevitable (at some point in one's life) as a consequence of simple genetic heritability (eg huntington's disease), a large number of medical conditions (including all behaviorial health disorders) are the expressed final pathway of a ...Read more


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Could gene therapy or transcranial Direct Current Stimulation (tDCS) or any other method help a patient with Williams syndrome?

Could gene therapy or transcranial Direct Current Stimulation (tDCS) or any other method help a patient with Williams syndrome?

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. ...Read more

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Is gene therapy an option for Down syndrome?

Is gene therapy an option for Down syndrome?

No: Someone with Down syndrome has an entire extra chromosome 21, this is incorporated into every single cell in the entire body, and causes creation of abnormal cell proteins and function. You cannot just pull out an entire chromosome - we are nowhere near that type of genetic therapy yet. Focus instead on what wonderful, full-loving people those with that extra chromosome are and enjoy them! ...Read more

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Is there any cure for hurler syndrome? And if there is then please tell me which doctor should I meet? And please tell me about gene therapy?

Is there any cure for hurler syndrome? And if there is then please tell me which doctor should I meet? And please tell me about gene therapy?

No: The is no cure for hurler syndrome, but some specialized medical centers are trying enzyme replacement and/or bone marrow transplantation therapy. One can search the web for medical centers involved in hurler syndrome treatment, and can start by looking at: http://marrow. Org/patient/disease_and_treatment/about_your_disease/metabolic_disorders/hurler_s_syndrome. Aspx. ...Read more

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Why isn't gene therapy used to produce insulin?

Why isn't gene therapy used to produce insulin?

Gene therapy: A good question but no immediate answer.

I am confident that within the next 5-10 years there will be major advances to produce insulin in your body. Till then you have to be patient.

PS: There may be some ongoing clinical trials in which you could participate. ...Read more

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Is cell therapy different from gene therapy?

Is cell therapy different from gene therapy?

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. ...Read more

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What are the complications with gene therapy?

What are the complications with gene therapy?

See: Here is some comprehensive information about gene therapy. Http://ghr. Nlm. Nih. Gov/handbook/therapy. Pdf ...Read more

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What disease is gene therapy is used to treat?

What disease is gene therapy is used to treat?

Selected: Gene therapy describes an approach whereby genetic technology will be used to treat many conditions. The easiest use of gene therapy is to replace a faulty gene. An example is glybera which is approved in europe to treat lipoprotein lipase deficienct, a rare genetic disorder. ...Read more

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What are the two approaches to human gene therapy?

What are the two approaches to human gene therapy?

Gene therapy: Here is some comprehensive information about gene therapy. Http://ghr. Nlm. Nih. Gov/handbook/therapy. Pdf ...Read more

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Help docs, is gene therapy available with hemochromatosis?

Help docs, is gene therapy available with hemochromatosis?

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. ...Read more

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

No: By late teens most kids are done growing because the growth plates fuse. There is no treatment to make growth plates open again once they've matured to fusion. ...Read more

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Can you tell me how can gene therapy on cysitc fibrosis sufferers work?

Can you tell me how can gene therapy on cysitc fibrosis sufferers work?

Gene therapy: Here is some comprehensive information about gene therapy. Http://ghr. Nlm. Nih. Gov/handbook/therapy. Pdf ...Read more

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

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

Pcr-FISH-karyotype: There are 3 main types of genetic tests. The old standard is to take a blood sample from the hip (marrow) and grow cells to look at all the chromosomes (karyotype). It is a 'broad strokes' look but not as sensitive as the others. A fish test looks at blood cells with 'probes' to the known genetic changes (moderately sensitive) and a PCR test is a 'molecular' test of the dna (very sensitive). ...Read more

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Can you tell me how are viruses genetically modified to be vectors for gene therapy?

Can you tell me how are viruses genetically modified to be vectors for gene therapy?

Gene therapy: Here is some comprehensive information about gene therapy. Http://ghr. Nlm. Nih. Gov/handbook/therapy. Pdf ...Read more

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How should gene therapy treat certain diseases?

How should gene therapy treat certain diseases?

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. ...Read more

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Need expert help here. What are 3 benefits of gene therapy?

Need expert help here. What are 3 benefits of gene therapy?

Info: Here is some comprehensive information about gene therapy. Http://ghr. Nlm. Nih. Gov/handbook/therapy. Pdf ...Read more

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I'm curious as to how gene therapy has been used to treat disease?

Gene therapy: Here is some comprehensive information about gene therapy. Http://ghr. Nlm. Nih. Gov/handbook/therapy. Pdf ...Read more

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Is the gene therapy for aids?

Is the gene therapy for aids?

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. ...Read more

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How does gene therapy cure diabetes?

How does gene therapy cure diabetes?

Possibly: I think the data is promising but not absolute. ...Read more

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Can you use gene therapy to treat cml?

No: "gene therapy" is not used as a treatment. However, the current treatments are designed to affect the genetic abnormality that leads to CML and are highly successful. ...Read more

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Pros of viral vectors in gene therapy?

They work!: For gene therapy to work, material must be transported into cells, such as new genes to insert, or proteins that cleave (break apart) chains or delete genes. Since viruses are capable of entering cells (that is how they infect them) and are able to integrate their own genes into the dna inside the cell, they can be effective in gene therapy. But the virus must be or be made harmless first. ...Read more

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Can gene therapy help with mitochondrial disorders?

Not yet: To date I have not heard of gene therapy applicable to mitochondrial disorders. ...Read more

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Can you tell me how gene therapy helps in curing various diseases?

Gene therapy: Here is some comprehensive information about gene therapy. Http://ghr. Nlm. Nih. Gov/handbook/therapy. Pdf ...Read more

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Is there any research being done to start gene therapy for scheuermann disease?

Check this site: Not sure, but www. Clinicaltrials. Gov has information on any studies that would be looking into this. Look at this link: http://clinicaltrials. Gov/ct2/results? Term=calve+disease (i just put in calve disease as a search term, to get broader results). Good luck! ...Read more

Dr. Fred McCurdy
63 Doctors shared insights

Williams Syndrome (Definition)

Neurodevelopmental disorder characterized by loquacious personality, abnormally sensitive hearing, supravalvular aortic stenosis, mental retardation, elfin facies, association with hypercalcemia due to abnormal sensitivity to vitamin d, idiopathic hypercalcemia of pregnancy. Due to deletion in elastin gene and probably several ...Read more


Gene (Definition)

A hereditary unit consisting of a sequence of dna that occupies a specific location on a chromosome and determines a particular characteristic in an organism. Genes undergo mutation when their dna sequence ...Read more