Gene Therapy Notes

FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling […]

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RISKS, PROBLEMS AND ETHICAL ISSUES OF GENE THERAPY

Gene therapy is used to deliver functional genes or therapeutic DNA into target cells and tissues of patients suffering from genetic disorders so that the defective genes in their body can be corrected, repaired or replaced with the normal genes. Though they may provide the only clue for treating some genetic diseases; gene therapy is

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APPLICATIONS OF GENE THERAPY IN TREATING MOLECULAR DISEASES

Molecular diseases (genetic disorders) are non-infectious inheritable diseases which are usually caused by mutations that alter the normal function of a particular gene. They are different from infectious diseases such as malaria and tuberculosis which are mainly caused by pathogenic microorganisms in that genetic disorders can be passed on from affected parents to their offspring’s

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POTENTIAL USES OF GENE THERAPY

Gene therapy techniques are an emerging field of experimental medicine and therapy for treating or preventing hereditary diseases and some infectious diseases. It works by altering the genetic instructions within an individual’s cells or genes by replacing the abnormal genes (responsible for the disease) with normal/healthy genes. In gene therapy, a gene that is missing

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IN VIVO DELIVERY FOR GENE THERAPY

In vivo delivery for gene therapy can also be called a direct delivery protocol for the transfer or introduction of a functional (therapeutic) gene to replace an abnormal gene in vivo. In vivo delivery for gene therapy is the gene therapy technique that delivers DNA, RNA or therapeutic protein directly into the cell or tissue

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PLASMIDS

Plasmids are extrachromosomal DNA molecules found in both eukaryotic and prokaryotic microorganisms that have the ability to replicate independently. In prokaryotes, plasmids are found in both Gram positive bacteria and Gram negative bacteria; and they usually range from 1.5 kb to 300 kb in size. In terms of the strands of their DNA molecule, most

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GENE THERAPY TARGETING AND DELIVERY

The efficient delivery of therapeutic proteins or DNA into specific cells or tissues of an organism to correct a mutant gene is paramount to the success of any gene therapy procedure; and the inserted therapeutic DNA or desired gene must be continuously expressed in vivo at appropriate physiological level in order to correct the mutated

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PREREQUISITES OR STEPS FOR GENE THERAPY

Gene therapy is an experimental discipline or research that uses functional gene (i.e. therapeutic DNA) to repair defective genes in genetic disease patients; and this growing field of molecular medicine is usually undertaken using modified nucleic acid molecules through a series of steps. Conventional medicine utilizes oral and parenteral drug administration as well as surgery

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