Question 1

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.

Question 2

how does gene therapy cure diabetes?

Accepted Answer

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

Question 3

pros of viral vectors in gene therapy?

Accepted Answer

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.

Question 4

can you use gene therapy to treat cml?

Accepted Answer

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.

Question 5

can gene therapy help with mitochondrial disorders?

Accepted Answer

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

Question 6

what are some of the major obstacles preventing the use of gene therapy in people?

Accepted Answer

FDA approval: The biggest obstacle before the widespread use of gene therapy in the USA is obtaining FDA approval.   A phase 3 trial is required prior to approval, treatment must show efficacy and minimal side effects compared to the disease being treated.  Phase 3 trials have had problems with immune system rejection of the wrapping material/virus for the gene therapy as well as issues with dosing..

Question 7

how do i know when will gene therapy become a reality for orphan diseases (single gene disorders)?

Accepted Answer

Glybera: The first gene therapy has now been approved in europe for an orphan disease. Several hundred therapies are currently in human clinical trials. So, i would say gene therapy is now a reality and the next 5 years will see a number of therapies approved.

Question 8

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 9

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 10

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.

Get Virtual Care

For Businesses

About Us

HealthTap Online Primary Care Doctors Accepting New Patients

Doctor answers about:

Gene therapy for albinism

A member asked:

Is the gene therapy for aids?

A member asked:

How does gene therapy cure diabetes?

A member asked:

Pros of viral vectors in gene therapy?

A member asked:

Can you use gene therapy to treat cml?

A member asked:

Can gene therapy help with mitochondrial disorders?

A member asked:

What are some of the major obstacles preventing the use of gene therapy in people?

A member asked:

How do i know when will gene therapy become a reality for orphan diseases (single gene disorders)?

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:

How should gene therapy treat certain diseases?

HealthTap Online Primary Care Doctors Accepting New Patients

Telemedicine

Primary Care

Manage Your Conditions

Women's Health

Children's Health

Men's Health

Senior Health

Wellness, Prevention & Lifestyle

Sexual Health

Mental & Behavioral Health

Travel Medicine

Urgent Care

Didn't find what you're looking for?

More of what we treat

Allergies

Antibiotics

Asthma

High Blood Pressure

Prescriptions

Type 2 Diabetes

For Businesses

Company

Health Content

Join Us

Download HealthTap

Questions?