gene therapy

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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HISTORY OF GENE THERAPY

The history of gene therapy techniques dates back to the early 1970s and 1980s when scientists (molecular biologists in particular) proposed the idea of using functional and therapeutic gene as medicines to treat genetic disorders in man. Though the idea received little support as at the time, gene therapy techniques is still in its experimental

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GENE THERAPY

Gene therapy is defined as the specific genetic manipulation and modification of an organism’s genome or genes through the delivery of therapeutic DNA or genes into host cells with little or no toxicity as a way of treating an inherited genetic disease or correcting a disorderly gene. Gene therapy techniques are basically used to correct

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NITROGENOUS BASES – Purine and Pyrimidines

Genes in the DNA code for proteins; and it is the gene that directs the cell in what particular order to assemble the amino acids which eventually becomes the building blocks of protein molecules. The cell of an organism must use one nucleotide or more in the DNA to spell out or specify each of

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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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