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E. Shyam P. Reddy: Promising Leap Toward Treating Alpha-1 Antitrypsin Deficiency

E. Shyam P. Reddy/LinkedIn

Jul 21, 2025, 14:57

E. Shyam P. Reddy: Promising Leap Toward Treating Alpha-1 Antitrypsin Deficiency

E. Shyam P. Reddy, Professor and Director of the Cancer Biology Program at Morehouse School of Medicine, shared a post on LinkedIn about recent article by Minjeong Kim, et al published in Nature Biotechnology:

“Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 antitrypsin deficiency—a promising leap toward treating humans with this rare inherited disease.

In a paper published in the journal Nature Biotechnology, researchers at UT Southwestern Medical Center introduced lipid nanoparticles—hollow, fatty spheres often 100,000 times smaller than the thickness of a piece of paper—that seek out and enter both lung and liver cells. This is significant because lipid nanoparticles tend to gather naturally in the liver versus other organs and tissues.

In mice genetically engineered with a mutation causing alpha-1 antitrypsin deficiency, these selective organ-targeting lipid nanoparticles delivered a payload of curative gene therapy, correcting about 40% of liver cells and 10% of lung cells affected by the disease. This also reduced levels of an abnormal protein associated with the genetic condition by over 80%.

‘[The researchers] are getting results that haven’t been gotten before,’ said Dr. Terence Flotte, a pediatric pulmonologist and dean of the University of Massachusetts T.H. Chan Medical School, who was not involved in the study. ‘It looks pretty darn convincing to me.'”

Title: Dual SORT LNPs for multi-organ base editing

Authors: Minjeong Kim, Eunice S. Song, Joseph C. Chen, Sumanta Chatterjee, Yehui Sun, Sang M. Lee, Shiying Wu, Priyanka Patel, Zeru Tian, Ariel Kantor, Brandon A. Wustman, David J. Lockhart, Daniel J. Siegwart

Read the Full Article on Nature Biotechnology

E. Shyam P. Reddy: Promising Leap Toward Treating Alpha-1 Antitrypsin Deficiency

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E. Shyam P. Reddy: Promising Leap Toward Treating Alpha-1 Antitrypsin Deficiency

‘[The researchers] are getting results that haven’t been gotten before,’ said Dr. Terence Flotte, a pediatric pulmonologist and dean of the University of Massachusetts T.H. Chan Medical School, who was not involved in the study. ‘It looks pretty darn convincing to me.'”