CRISPR Gene Editing can Treat Deadly Lung Diseases Before Birth

Highlights:

Surfactant is a lipoprotein mixture which is important for normal lung functioning
Mutations in the surfactant genes lead to fatal lung diseases like surfactant protein deficiency, cystic fibrosis and alpha-1 antitrypsin
Such fatal diseases can lead to immediate death after birth due to respiratory failure
Using CRISPR gene editing in-utero, scientists have edited out the harmful mutations
This proof-of-concept is a new beginning to deal with harmful lung diseases in fetuses before birth

CRISPR gene edits out a harmful mutation in surfactant genes which cause monogenic lung diseases. This gene editing tool can now help treat deadly lung diseases in fetuses before birth, reveals a new study.

Here's How CRISPR Gene Editing Treats Lung diseases

Surfactant is a lipoprotein mixture which is important for normal lung functioning. Mutations in the surfactant genes lead to fatal lung diseases like surfactant protein deficiency,

cystic fibrosis and alpha-1 antitrypsin. Such fatal diseases can lead to immediate death after birth due to respiratory failure with very few available therapeutic options.

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‘CRISPR can be used in-utero to edit lethal lung diseases in fetuses. This is a new beginning to deal with harmful lung diseases in fetuses before birth.’
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Using CRISPR gene editing in-utero, researchers at the Children's Hospital of Philadelphia (CHOP) have edited out the harmful mutations in animal models.

The lung is a suitable organ for targeted delivery to correct mutated genes. According to study co-leader, Professor Edward E. Morrisey of cardiovascular medicine at the Perelman School of Medicine, UPenn; the researchers were hoping to direct the gene-editing vehicles to target the cells lining the airways of lungs. William H. Peranteau, MD, study co-leader and pediatric and fetal surgeon at CHOP said that developing fetuses have some innate properties which make in-utero gene editing quite feasible.

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The research team conducted two rounds of experiments in mouse models. In the first round, they introduced CRISPR gene editing reagents into the amniotic fluid to target fetal lungs. The gene editing mechanisms were introduced into the developing fetus four days before birth which is equivalent to the human third trimester. The small size of the fetus and the immunological status makes it easier for gene editing as the amount of viral vector will be much less and the fetus will not be in a position to mount an immunological response to bacterial Cas9 proteins.

In the second experiment, the research team used gene editing to reduce the severity of congenital interstitial lung disease which is caused by a deficiency in surfactant protein C (SFTPC). Usually, all the mice born with this disease die of respiratory failure within a few hours of birth. However, the mice treated with prenatal gene-editing which inactivated the mutant SFTPC gene; showed an improvement in lung function and survival. Nearly 22 percent of the mice survived after this gene-editing intervention.

The team is hoping to move forward with further studies to evaluate better efficacy and efficiency of gene editing in the epithelial lining of lungs and to improve gene editing mechanisms.

Morrisey said that the team hopes to deliver diverse gene editing technologies to correct the exact mutations in genetic lung diseases in infants.

References:

In utero gene editing for monogenic lung disease - (https://stm.sciencemag.org/content/11/488/eaav8375)

Source: Medindia

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