What is a gene?
What is gene therapy?
What is a vector?
What is germline gene therapy?
What is somatic gene therapy?
What is the difference between in vivo and ex vivo gene therapy?
These are two different categories of somatic gene therapy. In ex vivo approach, the cells are obtained from the individual and are genetically modified/ corrected outside the body and then transplanted back into the same or different individual. In in vivo approach, the gene of interest is delivered directly to target cells/tissues, organs of the individual.
Why are viruses used in gene therapy?
Introduction of a gene into the cell requires a vehicle or agent to carry it, which is called a vector. Viruses are the preferred type of vectors/vehicles as they are very adaptable and efficient in delivering genetic material. These viruses have specialized mechanisms by which they can transfer their genetic material into the cells they infect. Types of viral vectors include adenoviruses, retroviruses, lentiviruses, adeno-associated viruses, herpes simplex virus etc.
What is the difference between gene therapy and stem cell therapy?
How does gene therapy work?
Gene therapy works by introducing therapeutic genes into a patient’s cells to replace missing or malfunctioning genes. Viruses are used as vectors or vehicles to transport genetic material inside the cells as they have a natural tendency to deliver nucleic acid cargo to target cell types. Prior to this, these viruses are modified to remove their ability to cause any infectious disease to the patient. The cells may be modified both inside (in vivo) and outside (ex vivo) the body.
What are genetic diseases?
Is gene therapy allowed in India?
What are the ethical issues associated with gene therapy?
The major ethical issues associated with gene therapy include: a) ethics on applicability of gene therapy: what is good and what is bad use of gene therapy (for example, it is ethical to treat a disease for which no curative measures exist, but not for enhancing abilities in normal, healthy individuals) b) ethics regarding suitability of a subject based on their disability type and severity for a particular kind of gene therapy c) ethics related to socio-economic factors which may limit accessibility of a therapy for the underprivileged.
What are the risks of gene therapy?
Gene therapy treatments may be associated with significant immune reactions which may supercede the beneficial effects of the therapy in certain cases. Since most gene therapy for monogenic diseases are long-term treatments, there may be specific unanticipated side effects of the therapeutic transgene itself in the long term.
What all diseases can be cured with gene therapy?
Gene therapy is currently being tested and approved for diseases which are not treatable by conventional, chemically synthesised or purified drugs/ biologics. These are primarily directed towards inherited genetic diseases or complex diseases without definitive cures such as certain types of cancers.
Is gene therapy a permanent cure?
Is gene therapy always successful?
How can one develop gene therapy in India?
For how long will one be disease free after undergoing gene therapy?
Why is gene therapy expensive?
Estimating the cost of gene therapy should include the cost savings due to avoiding multiple therapeutic sessions over time based on the current standard of care, and the related recovery potentials. The net-cost pricing need more studies for evidence to comment on net long term, over all costing, affordability and accessibility. Finally, producing the gene therapy “drug” is a technically challenging, laborious and time consuming process requiring a large input cost.
Can gene therapy treat cancer?
Gene therapy for cancer treatment is primarily in exploratory stage, although a few approved treatments are available such as Gendicine (for HNSCC) and a few types of CAR-T cell therapy. Such therapy includes genetically engineered viruses to directly kill cancer cells or gene transfer to cancer cells, and CAR-T cell based immunotherapy to find and kill cancer cells.
Is it a one-time treatment?
What is gene editing?
What is genetic enhancement?
At what stage of development is gene therapy in India?
What is CRISPR?
What is CAR-T cell therapy?
What is the risk of gene therapy?
What is eugenics?
Who was given the first gene therapy?
What's the difference between gene therapy and gene editing?
What are the three types of gene therapy?
At what stage of development is gene therapy in India?
Are any gene therapy research activities going on in India? In what way is the Government of India supporting these activities?
Are there any guidelines governing gene therapy research in India?
What is the need for such guidelines?
What kind of gene therapy research is covered by National Guidelines on Gene Therapy 2019?
Research for the diseases caused by genetic mutation for which conventional therapy includiResearch for the diseases caused by genetic mutation for which conventional therapy including small molecule drugs or alternative medicines is not available.ng small molecule drugs or alternative medicines are not available.
What is the mechanism of monitoring gene therapy research activities in the country?
Which act governs the use of gene therapy for treating patients?
What is a clinical trial?
Have there been any clinical trials pertaining to gene therapy in India? What are the results of those trials?
Who can conduct clinical trials? What are the requirements for the same?
How can I participate in a clinical trial? Do I have to pay in this process?