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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)

Gene therapy is also known as...

Gene therapy

Gene transfer therapy

Gene replacement therapy

Gene editing

Gene inhibition

What’s your Rare IQ?

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