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Home Industry News Inhalable gene therapy ‘could help pulmonary arterial hypertension patients’

Inhalable gene therapy ‘could help pulmonary arterial hypertension patients’

31st July 2013

A new study has revealed that the potential effectiveness of an inhalable gene therapy in reversing the damage caused by pulmonary arterial hypertension (PAH).

Investigators at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai demonstrated the benefits of gene therapy administered through a nebuliser-like inhalation device to restore the functions of the crucial enzyme SERCA2a in diseased lungs.

When SERCA2a is down-regulated, calcium stays longer in the cells than it should and induces pathways that lead to overgrowth of new and enlarged cells, but the tested therapy was able to completely reverse these effects among rat test subjects.

Study co-senior investigator Roger Hajjar of the Icahn School of Medicine at Mount Sinai said the results suggest "a gene therapy that is already showing great benefit in congestive heart failure patients may be able to help PAH patients who currently have no good treatment options".

In the UK, 4,000 to 5,000 people have PAH, with another 2,000 to 3,000 thought to be undiagnosed.ADNFCR-8000103-ID-801619188-ADNFCR

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