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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...

Leaving Groups

Leaving Groups

The nature of leaving groups strongly influences the outcome of a nucleophilic substitution reaction.
In general, in a nucleophilic substitution reaction, a nucleophile displaces a functional group, called the leaving group, from the substrate to give a substituted product. A leaving group departs the substrate molecule through heterolytic cleavage, taking the pair of electrons with it to become a relatively stable weak base in the form of an anion or a neutral molecule.
In a...

What are Biogeochemical Cycles?

What are Biogeochemical Cycles?

The most common elements in organic molecules, carbon, hydrogen, oxygen, nitrogen, sulfur, and phosphorus, are only available in the ecosystem in limited amounts. Therefore, these nutrients must be recycled through both biotic and abiotic components of the ecosystem, in processes generally called biogeochemical cycles.

The Water Cycle

The Water Cycle

The Earth’s hydrosphere includes all of the areas where the storage and movement of water occurs. Since water is the basis of all living processes, the cycling of water is extremely important to ecosystem dynamics.

The Citric Acid Cycle

The Citric Acid Cycle

The citric acid cycle, also known as the Krebs cycle or TCA cycle, consists of several energy-generating reactions that yield one ATP molecule, three NADH molecules, one FADH2 molecule, and two CO2 molecules.

The Phosphorus Cycle

The Phosphorus Cycle

Unlike carbon, water, and nitrogen, phosphorus is not present in the atmosphere as a gas. Instead, most phosphorus in the ecosystem exists as compounds, such as phosphate ions (PO43-), found in soil, water, sediment and rocks. Phosphorus is often a limiting nutrient (i.e., in short supply). Consequently, phosphorus is added to most agricultural fertilizers, which can cause environmental problems related to runoff in aquatic ecosystems.

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Gene Therapy Leaves a Vicious Cycle.

Reena Goswami¹, Gayatri Subramanian², Liliya Silayeva¹

¹Neuroscience Branch, Research Division, United States Army Medical Research Institute of Chemical Defense, Aberdeen, MD, United States.

Frontiers in Oncology

|May 10, 2019

Summary

This summary is machine-generated.

Gene therapy offers a one-time fix for genetic disorders by correcting errors in DNA. Recent advances in gene editing and viral vectors are revitalizing this "living drug" approach for various diseases.

Keywords:

clinical trials diseases and disorders gene therapy modern medicines viral vectors

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Area of Science:

Genetics
Molecular Biology
Biotechnology

Background:

Genetic mutations cause diseases, and current treatments are limited and often cause side effects.
Gene therapy, a "living drug," aims to correct genetic errors for a one-time cure.
Viral vector-based gene therapy has faced skepticism but is regaining interest.

Purpose of the Study:

To review viral vectors used in gene therapy.
To discuss advances and challenges in gene therapy.
To explore future perspectives for gene therapy.

Main Methods:

Review of current literature on gene therapy.
Analysis of viral vector technologies.
Examination of gene editing advancements.

Main Results:

Gene therapy offers a potential cure for genetic disorders by correcting DNA errors.
Advancements in gene editing and viral vectors have renewed interest in gene therapy.
Over 12 gene therapy drugs are now available for conditions like cancer and blindness.

Conclusions:

Gene therapy is a promising one-time treatment for genetic diseases.
Continued research in viral vectors and gene editing is crucial.
Gene therapy holds significant potential for treating a wide range of disorders.