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How does the gene therapy work?
Precisely, gene therapy is applied under two strategies; 1. Therapeutic gene strategy 2. Gene therapy for replacing a mutated gene Therapeutic gene strategy involves the insertion/administration of DNA that encodes a therapeutic protein packaged within a "vector", into the body. Inside the body, the DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patient's disease. Gene therapy for replacing a mutated gene involves the insertion of a functional gene (cloned into a vector) and administered into the body, where the functional gene express to neutralize the mutational effect.
What challenges are presented by gene therapies for central nervous system disorders?
Developing gene therapies for treating disorders of the nervous system poses unique challenges, such as how to introduce the therapeutic gene across the blood-brain barrier or how to target the therapeutic gene to one specific area of the brain.
Why should you use therapeutic cloning?
to clone a clincially important gene(to give protein)..eg:Insulin,glucocon,other factors
What can replace a gene in an animal's genome?
A new gene can be inserted into an animal's genome through genetic engineering techniques, such as gene editing or transgenesis. These techniques can replace a faulty gene with a functional one, or introduce a completely new gene into the genome. Additionally, gene therapy can be used to deliver therapeutic genes into an animal's cells to treat genetic disorders.
What is gene silencing?
Gene silencing is a process by which the expression of a specific gene is suppressed, leading to a reduction or elimination of the gene's protein product. This can occur through various mechanisms, such as RNA interference or epigenetic modifications, which can silence gene expression at the transcriptional or post-transcriptional level. Gene silencing plays a crucial role in regulating gene expression and can be used in research and therapeutic applications.
What obstacles must gene therapy overcome?
Gene therapy must overcome challenges such as efficiency in delivering therapeutic genes into target cells, avoiding off-target effects, ensuring long-term expression of the therapeutic gene, immune response issues, and the potential for unintended consequences such as mutations or cancer. Additionally, the high cost of gene therapy treatments and regulatory hurdles pose significant obstacles to its widespread adoption.
What microbes are used with gene therapy in duchenne muscular dystrophy?
Adenoviruses and adeno-associated viruses (AAVs) are commonly used in gene therapy for Duchenne muscular dystrophy (DMD). These viruses are used as vectors to deliver the therapeutic gene to muscle cells to replace the mutated gene responsible for DMD. Additionally, lentiviruses have been investigated as potential vectors for gene therapy in DMD.
How many ways of cloning are there?
There are three main ways of cloning: gene cloning, reproductive cloning, and therapeutic cloning. Gene cloning involves replicating specific genes or DNA sequences, while reproductive cloning aims to create an identical copy of an organism. Therapeutic cloning is used to create stem cells for medical purposes.
Why does gene therapy not always work?
Gene therapy may not always work due to challenges in delivering the therapeutic gene to the target cells, potential immune responses, difficulty in achieving sustained gene expression, and the complexity of genetic diseases. Additionally, off-target effects, limited understanding of gene function, and ethical concerns surrounding genetic manipulation can also impact the success of gene therapy.
How do you produce therapeutic proteins by plants?
Therapeutic proteins can be produced in plants by introducing the gene encoding the protein of interest into the plant's DNA. The plant then produces the protein during its growth and development. The therapeutic protein can be extracted from the plant and purified for use in medical treatments.
Difference between therapeutic and non therapeutic technique?
Therapeutic is an immediate physical treatment, non-therapeutic is a treatment such as sterilisation
What must happen inside the cell for gene therapy treatment to be successful?
For gene therapy to be successful, the therapeutic gene must be delivered into the target cells and integrated into the cellular DNA. The gene should be expressed and produce the functional protein to correct the underlying genetic disorder. The protein should function properly and restore normal cellular function.
