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What exactly is gene therapy?

Genetic engineering has revolutionized the way scientists think about diseases. New technological advancements like gene editing have allowed medical researchers to alter the composition of DNA, a groundbreaking technique that has led to the development of gene therapy. 

What is gene therapy?

Gene therapy is a novel treatment method that uses genetically engineered medicine to treat genetic diseases. Human gene therapy is performed using functioning genes able to counteract the effects of mutated genes that cause genetic disorders.

The development of gene therapy products requires the administration of particular DNA or RNA sequences. Specific genes are carefully selected and prepared to modify defective genes. This novel therapeutic approach offers potential new treatments for a variety of diseases including many hereditary diseases like muscular dystrophy and cystic fibrosis, viral infections, diabetes, hemophilia, AIDS, and various forms of cancer. 

Considerable efforts are being made by medical researchers to enhance the safety and effectiveness of gene therapy techniques. Scientists expect that in the near future gene therapy can be used to insert genetic material directly into a patient’s cells, reducing the need for drug products and surgery. 

What is the difference between gene therapy and cell therapy?

Both cell and gene therapy can potentially be used to treat genetic and acquired medical conditions. However, cell therapy is used to transfer cells into the body, while gene therapy transfers genetic material instead.

What is cell therapy?

Cell therapy is a treatment method that modifies cells in order to improve the immune system or cure diseases. Cells from patients are usually substituted with adult or fetal stem cells which possess the ability to enhance cell maturation and tissue renewal. Stem cells are extremely flexible and can be turned into specialized tissue cells. 

Human cells from umbilical cord blood and similar tissues can be used to develop treatments for several diseases. Fetal cells offer more therapeutic advantages over adult cells, as they divide more and are better suited to transform into the desired outcomes. For instance, the liver stem cells of a fetus will produce much more bone than adult liver cells during bone marrow transplantation.

Risks of cell therapy

Keeping cell division under control is a potential challenge of cell therapy methods. Stem cells have a growth advantage in the body they are transplanted into, which may lead to them growing uncontrollably and developing into a form of cancer or teratoma. Nonetheless, this phenomenon is rarely seen in stem cell transplantation patients. Patients are strongly encouraged to ask their doctor about the potential risks of any treatment they wish to adhere to. 

What is gene therapy used for?

The goal of gene therapy is to alter faulty genes inside a body’s cells in order to treat and stop ailments. By replacing damaged genetic material with healthy genes, a wide range of diseases may be handled. 

Researchers acknowledge the following as the main applications of gene therapy: 

  • Replace defective genes: When certain genes stop working or begin behaving incorrectly, the cells that host them become defective. For example, a gene called p53 is responsible for stopping tumor growth. If it malfunctions, the body becomes at risk of developing cancer. By replacing the p53 gene with a healthy variant, doctors can trigger the death of cancerous cells.
  • Mend mutated genes: Gene therapy can be used to turn off mutated genes responsible for promoting diseases. Likewise, healthy genes that have stopped working can be kickstarted back to action so they can inhibit the diseases.
  • Enhance immune responses: Sometimes the immune system doesn’t fight diseased cells because immune cells are unable to recognize them as threats. Gene therapy can be used to make diseased cells more evident. This approach allows a doctor to train a patient’s immune system to recognize the hazardous potential of faulty cells. 

How to become a candidate for gene therapy

The most accessible way for a patient to become a candidate for gene therapy is by participating in clinical trials. These are specialized research studies that help doctors devise the overall safety of new drugs and treatments. Gene therapy trials are also used to better understand the effects gene therapy has on the body. 

During a clinical trial, patients may have their blood drawn or have part of the bone marrow from their hipbone removed using a large needle. Cells found within the gathered genetic material are then exposed to a vector (usually a viral vector) in a lab setting. The vector being used contains functional genes which are transferred to the cells. The cells are then injected back into the body through a vein or into tissue, taking the vector along with them.

While viral vectors are the most common vectors used to carry altered genes into a patient’s body, other types of vectors have also caught the attention of medical researchers. Stem cells and liposomes, for instance, can be used to fight disease and carry new therapeutic genes. The latest technological advancements allow for plasmid DNA therapy, where plasmids created in a lab are used to directly insert pharmaceuticals into the body.

What is plasmid DNA?

What are the 2 types of gene therapy?

Somatic gene therapy

Somatic gene therapy is performed by inserting therapeutic DNA into a patient’s body cells. Somatic cell genes used in this therapeutic approach are created via genome editing, a set of techniques and technologies designed to alter an organism’s DNA. 

In order for somatic gene therapy to be successful, the desired genes have to be expressed. Additionally, this new expression must be regulated to prevent a disease triggered by over-expression.  

Germline therapy

This method involves the use of the genes inside germ or gamete cells, taken from tissue derived from reproductive cells (ova and sperm). The purpose of germline therapy is to make changes to DNA that can be passed on to future generations. Germline therapy promises to revolutionize the treatment of genetic disorders by eliminating their existence from the gene pool. 

Because the potential risks of germline theory are unknown to modern science, it has dramatically divided healthcare professionals’ opinions towards it. Because it targets reproductive cells, unintended mutations may be introduced as a by-product of the therapy. Germline therapy also poses the ethical question of whether an individual should have their genetic material altered without their consent. 

The jurisdictions of several countries including Germany, Switzerland, and Australia have prohibited the use of germline therapy. The US National Human Genome Research Institute, however, considers that ethical arguments should be accompanied by new genetic modification practices (source). The practical use of germline theory is also limited due to its elevated cost. 

How does gene therapy work?

The specific gene therapy procedure a patient will be subjected to and the time it will take vary depending on the disease being treated and the chosen type of gene therapy. Some therapy approaches are personalized, using a patient’s cells’ genome to treat them. 

There are 4 main steps that can be identified in the development of a gene therapy procedure:

Step 1: Consultation

During the consultation process, patients should discuss the potential risks and benefits of the different treatment options with their doctor. This process may include multiple visits over an extended time period, where the patient’s condition is assessed and the decision of whether to follow a particular treatment or not is made. A patient’s eligibility for gene therapy is chosen during this stage. 

Step 2: Preparation

For gene therapy to commence, the cells of a patient may have to be collected. If a personalized method of gene therapy is being developed, then the drug manufacturer might be involved during this procedure. Patients will be guided through this process by their doctor, who will give instructions for any preparative measures a patient may have to undertake.

Step 3: Treatment

A gene therapy treatment may be an inpatient procedure, so patients should be ready to spend the night in the hospital. It is possible that conditioning with chemotherapy may have to be included as part of this stage. Naturally, gene therapy is performed by a specialist together with a specialized care team.

Step 4: Recovery

After a physician consultation and some recovery time, a patient will be discharged from treatment. Follow-up appointments to gauge the effectiveness of the treatment may be included, as well as home healthcare needs for some time. A patient may see the need to enroll in a registry to monitor the long-term results of their treatment.

Valentia Analytical services 

Valentia Analytical is an industry leader in the field of pharmaceutical development. By focusing on the needs of patients, Valentia Analytical can handle data in a time-effective manner and deliver the highest quality results. 

The commercialization of pharmaceutical products requires substantial amounts of research and development and the proper facilities to handle experimentation. Thanks to the expert minds behind Valentia Analytical, the laboratory workflow is kept in pristine condition, winning the praise of customers and regulatory inspectors alike. 

Valentia Analytical offers a wide range of services to aid in the development of therapeutic solutions. These include: 

  • Gene Therapy: Plasmid DNA may be characterized for topoisometric purity and other characteristics using techniques of HPLC, CE (Capillary Electrophoresis), and SDS-PAGE.  Plasmid DNA purity may also be analyzed by using the SoloVPE application.  
  • Biotechnology: Proteins with monoclonal antibodies can be identified and tested. Samples can be gathered from various sources including cell media, raw materials, and drug products. 
  • Pharmaceutical development: The purity, potency, identity, and impurity content of small molecules can be tested.
  • Combinational medical devices: Medical equipment designed to deliver drugs or proteins can be tested for various characteristics, including drug load and chemical impact by sterilization.

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