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Research

Our Commitment

 

Since 1995, the Alpha-1 Foundation has funded over $65 million to find a cure for Alpha-1.

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Chart Investment

Basic Researchplus

  • Molecular biology of Alpha-1 Antitrypsin (AAT) expression
    • Mechanisms of AAT synthesis, folding and secretion
    • Molecular pathology of Z AAT gene expression
  • Animal Model Development
    • Development of an AAT deficient animal model for lung
    • Development of an AAT deficient animal model for liver
  • Lung-Focused Research
    • Determinants of lung growth, turnover, maintenance and regeneration
    • Mechanisms of tissue destruction, response to injury, and inflammation
  • Liver-Focused Research
    • Determinants of liver growth, maintenance, turnover and regeneration
    • Mechanisms of hepatocellular toxicity and liver damage
  • Technology Development
    • Hepatocyte transplantation
    • Gene therapy: enhancement, replacement, extinction and repair
    • Epigenomics of Alpha-1 gene regulation
    • Small molecule anti-proteases
    • Small molecule, high through-put library screening
  • Stem Cell Research and Regenerative Medicine
    • In vitro disease modeling, drug screening, and personalized medicine
    • Stem cell-based therapies
    • Lung and liver tissue development and engineering artificial organs

Clinical Researchplus

  • Epidemiology and Natural History of AAT deficiency
    • Impact of primary care and allied health care providers in detecting alpha-1
    • Prenatal and newborn screening pilot studies
    • Predicting course and outcomes
    • Defining the risk of clinical manifestations in heterozygote carriers
      • Longitudinal study of PI*MZ individuals
    • Natural history following lung transplantation
    • Natural history of lung disease following liver transplantation
  • New diagnostic technologies
  • Modifier genes or epigenomic influences affecting Lung and Liver in AAT deficient individuals
  • Transcriptomic and proteomic correlates of lung disease progression or stability
  • Role of inflammation in the pathogenesis of AAT lung disease
  • Establishment of effective clinical outcomes measures in AAT deficiency
    • Biomarkers of early lung or liver disease or of disease exacerbations
    • MRI, Quantitative CT scanning, fibroscan or other new modalities to assess lung or liver disease progression and response to therapy
  • Quality of life, patient reported outcomes, healthcare utilization, and symptom management
  • Environmental modifiers of lung and liver disease in AAT deficient individuals
    • Microbiome as a potential disease modifier
    • Gene-environment interactions
  • Clinical manifestations of AAT Deficiency other than in the lungs and liver

Novel Therapiesplus

  • Alpha-1 antitrypsin augmentation therapy
    • Development of recombinant therapy approaches
    • Development of aerosolized AAT therapy
    • Determining the utility of AAT therapy in deficient lung transplant recipients
    • Therapeutic dose analysis
  • Improving outcomes in lung and liver transplant recipients
    • Use in post-transplant rejection
  • Treatment of pathophysiological manifestations (e.g., hyperinflation, cirrhosis, cholestasis) of AAT deficiency in lung and liver
  • Anti-inflammatory therapy
  • Small molecule antiprotease therapy
  • Cell-based therapies
  • Gene therapies
    • Gene replacement therapy
    • Enhancing or silencing AAT expression
  • Chemical chaperone therapy
  • Biomarkers as an index of therapy in lung and liver

Ethical, Legal, Social Issuesplus

  • Newborn testing/screening
  • Targeted detection
  • Social dimensions of living with A1ATD
  • Equitable access to, and distribution of, medical therapies
  • Impact of genomic information on Alpha-1 patients and their families
  • Impact of interventions on patient adherence and emotional well-being
  • Impact of Alpha-1 on families

Detectionplus

  • Increasing AATD testing in primary care
  • Screening and diagnosis of AATD through electronic health records
  • Alpha-1 Antitrypsin Deficiency Pilot and Feasibility Screening and Detection Study in a Targeted Population
  • Newborn Screening
Patient Research Registry

The Alpha-1 Foundation Research Registry is the most impactful engagement a patient can make to take an active role in finding a cure for Alpha-1.

You may also view trials that are recruiting patients here.

Research Grant Opportunities

The Alpha-1 Foundation offers research opportunities for both in-cycle and out-of-cycle timelines.

Patients Needed
for Critical Study

A five-year, multi-center study of Alpha-1 liver disease in adults has enrolled its first participants and is actively recruiting Alphas to take part, says Alpha-1?

The Alpha-1 Project (TAP)

The Alpha-1 Project (TAP) provides critical funding and advice to speed up the commercialization of therapies for Alpha-1 Antitrypsin Deficiency.