Alpha 1 Antitrypsin Deficiency News and Research

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Lung disease patients more likely to develop heart disease independent of any risk factors

Patients with lung disease are more likely to develop heart disease independent of any risk factors, according to new research.

22 Mar 2024

Augmentation therapy can save lives of people with alpha-1 antitrypsin deficiency, study shows

Boosting levels of a deficient protein has clear survival benefits for people with the genetic condition alpha-1 antitrypsin deficiency (AATD), according to new research led by RCSI University of Medicine and Health Sciences.

1 Nov 2023

Huntington's disease impairs autophagy in older patients

Huntington's disease, a fatal, inherited neurodegenerative condition, is caused by a genetic error present at birth, though its symptoms often don't begin until middle adulthood.

28 Oct 2022

Insights into molecular folding of AAT protein will help develop new therapies for alpha-1 antitrypsin deficiency

In a major new development in the quest to develop better gene therapies with which to treat a host of diseases, researchers at the University of Massachusetts Amherst and UMass Chan Medical School recently announced that they have mapped the expression and maturation of the protein alpha-1 antitrypsin (AAT) with unprecedented clarity.

25 Jul 2022

People diagnosed with alpha-1 antitrypsin deficiency more likely to stop smoking, study shows

New research shows that people diagnosed with a genetic condition, called alpha-1 antitrypsin deficiency (AATD), are far more likely to stop smoking and therefore prevent the development of lung disease.

3 Mar 2021

Aprotinin inhibits SARS-CoV-2 entry and replication in cells cultures

Researchers at the University of Kent in the UK and the Hannover Medical School and Goethe University in Germany have observed that the protease inhibitor aprotinin can inhibit viral replication by preventing SARS-CoV-2 entry into host cells.

24 Nov 2020

Scientists describe genetic program behind primordial lung progenitors

For the first time, researchers describe the genetic program behind primordial lung progenitors--embryonic cells that give rise to all the cells that form the lining of the respiratory system after birth.

31 Jan 2020

Researchers use new technique to improve the ability to model lung diseases

Using a combination of pluripotent stem cells (cells that can potentially produce any cell or tissue type) and machine learning (artificial intelligence that allows computers to learn automatically), researchers have improved how they generate lung cells.

30 Jan 2020

Alnylam submits CTA application to MHRA to initiate Phase 1/2 study of ALN-AAT02

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that it has submitted a Clinical Trial Authorization application to the Medicines and Healthcare products Regulatory Agency to initiate a Phase 1/2 study of ALN-AAT02, an investigational RNAi therapeutic targeting alpha-1 antitrypsin for the treatment of AAT deficiency-associated liver disease.

3 Oct 2018

Arrowhead completes dosing of ARO-AAT Phase 1 study to treat rare genetic liver disease

Arrowhead Pharmaceuticals Inc. today announced that it has completed dosing of a Phase 1 clinical study of ARO-AAT, the company's second generation subcutaneously administered RNA interference therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency.

31 Aug 2018

Study helps to better understand disease caused by Alpha-1 antitrypsin deficiency

The disease consists of a production deficit of Alpha-1 antitrypsin, which is produced in the liver. The main role of this protein is to protect the lungs from degradation or inflammation caused by infections that attach lung tissue, as well as from external agents such a tobacco or pollution.

14 Aug 2018

Texas Biomedical Research Institute tests promising new treatment for HBV infection

Research at the Southwest National Primate Research Center on the campus of Texas Biomedical Research Institute helped advance a new treatment now in human trials for chronic hepatitis B virus (HBV) infection.

7 Nov 2017

Opportunities and risks of direct-to-consumer genetic health risk tests

Accurate genetic testing stands to transform modern medicine by offering effective, personalized treatment.

13 Apr 2017

FDA authorizes marketing of first direct-to-consumer GHR tests for certain diseases

The U.S. Food and Drug Administration today allowed marketing of 23andMe Personal Genome Service Genetic Health Risk (GHR) tests for 10 diseases or conditions.

6 Apr 2017

Exosome isolation using ultracentrifugation

In the outpatient clinic and lab, our research studies aim to understand biological functions of exosomes and membrane particles.

From Beckman Coulter Life Sciences - Centrifugation 9 Feb 2017

RNA silencing technique developed by spinoff from UM-Madison effective in battling hepatitis B

A method for "silencing" RNA that emerged from a University of Wisconsin-Madison spinoff company is in clinical trials in Europe, Asia and the United States against hepatitis B, an infection that can destroy the liver.

11 Aug 2016

New lung-targeted gene therapy shows promise in improving treatment for emphysema

Researchers have developed a new strategy using lung-targeted gene therapy that may lead to improved treatments for inherited diseases including emphysema.

6 Jul 2016

Innovative approach to treating AAT deficiency

Researchers have demonstrated the feasibility of delivering an RNA that encodes for the protein alpha-1-antitrypsin (AAT)--which is missing or nonfunctional in the genetic disorder AAT deficiency--into cells in the laboratory, enabling the cells to produce highly functional AAT.

28 Jul 2015

Therapy for genetically-caused emphysema slows progression of lung disease

A landmark clinical study in the Lancet provides convincing evidence that a frequently overlooked therapy for genetically-caused emphysema is effective and slows the progression of lung disease.

29 May 2015

Alnylam files clinical trial application for alpha-1 liver disease, presents data at DDW

Alnylam Pharmaceuticals, Inc. has announced that it has filed a Clinical Trial Application (CTA) with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial with ALN-AAT, a subcutaneously administered investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease (alpha-1 liver disease).

19 May 2015