Viral or non viral gene therapy, in brain

In summary, the conversation discusses the potential negative health effects of using viral gene therapy to add extra copies of a gene in the human brain. It also raises questions about the effectiveness of non-viral gene therapy and the likelihood of negative effects occurring with different types of viruses. One negative effect mentioned is the need for stereotaxic neurosurgery for delivery of the virus to the brain, and another is the generation of antibodies to the viral capsid proteins. However, overall, viral gene therapy to the brain is seen as a promising platform.
  • #1
bioquest
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Im not sure if anybody here can answer this, but, this is my question

What negative health effects etc can occur from viral gene therapy being used on a human brain (to add one or more extra copies of a gene)

what are the odds of those negative effects occurring in the person from viral gene therapy being used on a human brain? (To add one or more extra copies of a gene)

Would non viral gene therapy in a human brain be effective enough to add one or more extra copies of a gene?

These questions are in regards to each type etc of virus though, not just AAV.

thanks..not sure if this is the area of expertise of anyone here but anyways
 
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  • #2
one negative effect is delivery of virus to the brain that would most likely involved stereotaxic neurosurgery. another negative effect is antibodies being generated to the viral capsid proteins but i think you will probably be OK with the first administration, so as long as you have long-term expression from your viral vector you will not have to worry about a second administration. viral gene therapy to the brain is a promising platform.

non-viral gene therapy sucks (i assume you are talking about plasmid DNA).
 

1. What is viral gene therapy and how does it work?

Viral gene therapy is a type of treatment where a virus is used to deliver genetic material into a cell. The virus is modified to carry the desired genetic material, which is then delivered into the cell's DNA. This allows for the insertion of new genetic information, which can potentially correct genetic disorders or improve cellular functions.

2. What is non-viral gene therapy and how does it differ from viral gene therapy?

Non-viral gene therapy is a type of treatment where non-viral delivery methods, such as liposomes or nanoparticles, are used to transfer genetic material into cells. This method does not use viruses, which can decrease the risk of immune reactions and the potential for viral mutations. However, non-viral gene therapy is less efficient in delivering genetic material compared to viral gene therapy.

3. Can gene therapy be used to treat brain disorders?

Yes, gene therapy can be used to treat brain disorders. In fact, it has shown promise in treating disorders such as Parkinson's disease, Alzheimer's disease, and Huntington's disease. The delivery of genetic material directly into the brain can potentially correct genetic mutations or enhance brain cell function.

4. What are the potential risks and side effects of gene therapy in the brain?

Some potential risks and side effects of gene therapy in the brain include immune reactions, off-target effects, and the potential for the introduced genetic material to cause unintended changes in the patient's DNA. There is also a risk of the virus used in viral gene therapy mutating and causing unintended effects. Additionally, the delivery method itself can cause damage to brain tissue.

5. What are the current limitations of gene therapy in the brain?

One of the main limitations of gene therapy in the brain is the difficulty in delivering genetic material to specific areas of the brain. The blood-brain barrier, which protects the brain from foreign substances, can also prevent the delivery of therapeutic agents. Additionally, the long-term effects of gene therapy in the brain are still being studied, and there may be unknown risks associated with this treatment method.

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