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Gene Therapies Write for Us

<yoastmark class=Gene Therapies Write for Us – Human gene therapy seeks to modify or manipulate gene expression or alter biological characteristics of living cells for therapeutic use gene therapy is a technique that modifies a person’s genes to treat or cure a disease gene therapies can act by several mechanisms:

  • Replacing a disease-causing gene with a healthy copy of the gene
  • Disrupting a disease-causing gene that is not working properly
  • Introducing a new or modified gene into the body to help treat a disease
  • Gene therapy products are being studied to treat cancer, genetic, and infectious diseases.

There are Different Types of Gene Therapy Products, Including:

  • Plasmid DNA: Circular DNA molecules that can be genetically modifi to transfer therapeutic genes into human cells.
  • Viral vectors: Viruses can naturally deliver genetic material into cells, and thus, some gene therapy products are derive from viruses. Once viruses have been modified to remove their ability to cause infectious disease, these modified viruses can be used as vectors (vehicles) to transfer therapeutic genes into human cells.
  • Bacterial vectors: Bacteria can be modified to prevent them from causing infectious disease and then used as vectors (vehicles) to transfer therapeutic genes into human tissues.
  • Human Gene Editing Technology: Gene editing aims to inactivate harmful genes or repair transgenes.
  • Patient-derived cellular gene therapy products: Cells are removed from the patient, genetically modified (often with a viral vector), and then returned to the patient.

Gene therapy products are biological products regulated by the Food and Drug Administration’s Center for Biological Evaluation and Research (CBER). Clinical studies in humans require an investigational new drug (IND) application to be submitted before clinical studies begin in the United States. Marketing a gene therapy product requires submission and approval of a Biology Licensing (BLA) application.

Understanding Genes

Genes come from our parents. They dictate everything from the color of our eyes to our immune system to our susceptibility to disease. Genes contain the information needed to build and maintain cells by encoding proteins.

A gene is a small piece of a cell’s DNA comprising four chemicals called bases. These bases make up the backbone of DNA and — much like guidebooks — provide instructions for the assembly of proteins, the building blocks of the human body, such as muscles, organs, and the immune system.

The problem is that genes aren’t always built right. When this happens, it changes the DNA of the gene. This is called a mutation. Mutations can be passed on from someone’s parents or occur for the first time in that person. A mutation in a gene can change the way proteins work. Sometimes, mutations can cause disease. This is where gene therapy may help.

How Does Gene Therapy Work?

Add genes

  • Genetic addition is used to treat conditions caused by a variation in one gene.
  • This one-time treatment involves introducing a functional copy of the missing or defective gene into a person’s cells via a viral or nonviral genetically modified vector (delivery vehicle).
  • The goal of this treatment is for the new gene to produce a protein that the body was (previously) unable to produce enough of.

Gene Silencing Symbol

  • Post-transcriptional gene silencing inactivates messenger RNAs (mRNAs) to prevent gene expression.
  • The mRNA of a specific gene, which would usually be expressed (turned on), is suppressed (knocked down).
  • When knocked down, mRNA no longer produces an active protein at the same level as before silencing.
  • Gene silencing methods fight cancer and other conditions like sickle cell disease.

Gene Editing Symbol

  • Gene editing is a precise technique used to add, remove or alter specific sections of DNA.
  • It involves making cuts in the DNA at the exact location where it is broken.
  • After cutting the DNA, the cell naturally repairs itself.

By disrupting unwanted sequence elements or by copying and pasting in new sequences to fix defective sequences, gene editing can lead to permanent.

Genetic changes. Cells can function normally and reverse disease processes.

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