Journal of Genetic Syndromes & Gene Therapy
Open Access

3^rd Annual Congress on Rare Diseases and Orphan Drugs

October 30-November 01, 2017 San Antonio, USA

Scientific Tracks Abstracts

• The use of CRISPR/Cas9 to treat hereditary diseases: Duchenne muscular dystrophy, Friedreich’s ataxia and Alzheimer disease

  Jacques P Tremblay, Khadija Cherif, Dominique L Ouellet, Benjamen Duchene, Jean-Paul Iyombe, Catherine Gerard, Joel Rousseau and Antoine Guyon

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• Integrating informational resources for drug repurposing in rare diseases

  Maria Shkrob, Matthew Clark, Anton Yuryev and Rick Thompson

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• L-cystine diamides as a novel therapy for cystinuria

  Amrik Sahota, Min Yang, David Goldfarb, Michael Ward, Jay A Tischfield and Longqin Hu

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• Using quantitative pharmacology to overcome challenges in hematopoietic cell transplantation: Application towards primary immune deficiencies and inborn errors of metabolism

  Janel Long-Boyle

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• Nephrotic syndrome: Predicting and defining therapeutic outcomes by systems biology omics approaches

  Shipra Agrawal

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• Porphyrias - An update on the management and unmet clinical needs

  Ashwani K Singal

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• The Middle East: A mine for orphan drugs development

  Tony Zbeidy

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• Pharmacological chaperone therapy for neuronopathic Gaucher disease

  Aya Narita

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• Modeling Li-Fraumeni syndrome by induced pluripotent stem cells

  Dung-Fang Lee

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• Genomic approaches in modern biotechnology: From discovery, translation to implementation

  David Dongliang Ge

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• A case of Hoffman′s syndrome masquerading as pituitary tumor

  Arnab Ghosh and Manish Bhartiya

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• Cutaneous infarcts and antineutrophil cytoplasm antibodies positive vasculitis in angioimmunoblastic T-cell lymphoma: A rare presentation

  Abhish Mohan, Sandeep Kumar and Pankaj Puri

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• POEMS syndrome in a 65 years old lady presenting with peripheral neuropathy and recent onset diabetes

  Arnab Ghosh and Sanjeevan Sharma

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