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Gene therapy

Gene therapy is a medical technology for treating or preventing disease by correcting the underlying genetic problem. Gene therapy techniques allow the treatment of genetic-based disorders by altering the genes that cause diseases

5 Facts you should know

FACT

1

Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use

FACT

2

Somatic gene therapy represents mainstream clinical research, in which a gene (therapeutic DNA) is used to treat disease

FACT

3

The first therapeutic use of gene transfer was performed by French Anderson in a trial starting in September 1990

FACT

4

In 2017, Spark Therapeutics' Luxturna for RPE65 mutation-induced blindness was the first therapeutic gene therapy approved in the United States

FACT

5

To date, most of the successful approaches in gene therapy utilize adeno-associated viruses (AAVs)

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Gene therapy is also known as...

Gene therapy

Gene transfer therapy

Gene replacement therapy

Gene editing

Gene inhibition

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Which type of viral vector is the most commonly used gene therapy delivery system in current clinical trials?

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