Rare Infectious Disease News

5 Facts you should know about

Gene therapy

fivefacts-light.svg

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

2

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

3

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

4

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

5

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

Rare Infectious Disease News