19K views · 1.3K reactions | @muellerlab at UMass #Medical School has developed a dual function approach using AAV vectors targeting both #lung and #liver diseases in alpha-1 antitrypsin deficient (AATD) patients. https://www.umassmed.edu/muellerlab/ | Sci Ani | Facebook

Alpha-1 Antitrypsin Deficiency is a genetic disorder that can affect the lungs, and/or liver, and skin. I was diagnosed in 2018, but did not receive the appropriate care until 2021. This is not uncommon…Alpha-1 is rarely diagnosed; and often, it is found late in the disease progression. Because the medical community is generally uninformed, treatment can be delayed or denied which leaves patients without medical care or following obsolete protocols. Alpha-1 is gaining recognition though and hope

Treatment of Alpha-1 Antitrypsin Deficiency in children include...