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Spotlight on Gene Therapy: finding a Cure

Mueller Lab announces presentations at upcoming ASGCT meeting

Friday, April 28, 2017
By:  Florie Borel

The Mueller Lab for Gene Therapy announces that 5 abstracts have been accepted for presentations at the upcoming ASGCT meeting.

Christian Mueller, PhD, said “We are making steady progress in both our gene editing research programs to treat Alpha-1 Antitrypsin Deficiency and Amyotrophic Lateral Sclerosis, furthermore we have completed the IND-enabling studies for our gene silencing program for SOD1-linked ALS and we are looking forward to our ‘first in human’ use of this therapy.”

 ASGCT 2017

Specific details on the presentations at ASGCT include:

  • Title: Proof-of-Concept for Non-Nuclease-Mediated Genome Editing to Treat Alpha-1 Antitrypsin Deficiency
    Oral Session Title: Cardiovascular and Pulmonary Gene and Cell Therapies
    Date and Time: Wednesday, May 10, 2017, 5.00 p.m.
    Location: Delaware AB


  • Title: Artificial miRNAs Reduce Human Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington’s Disease
    Oral Session Title: Oligonucleotides Therapeutics
    Date and Time: Wednesday, May 10, 2017, 5.00 p.m.
    Location: Maryland ABC


  • Title: Editing out Five Serpina1 Paralogs to Create a New Mouse Model of Genetic Emphysema (Poster 248)
    Oral Session Title: Novel Models for Genetic, Metabolic and Endocrine Disorders
    Date and Time: Wednesday, May 10, 2017, 5.30 p.m. - 7.30 p.m.
    Location: Exhibit Hall A & B South


  • Title: Silencing SOD1 to Treat Amyotrophic Lateral Sclerosis: Preclinical Studies in Cynomolgus Monkeys
    Oral Session Title: Preclinical Progress Towards Therapies for Neurologic Disorders
    Date and Time: Friday, May 12, 2017, 5.00 p.m.
    Location: Delaware AB


  • Title: Gene Editing ALS Causing (GGGGCC)n Repeat Expansion in C9orf72 Using CRISPR/Cas9 System (Poster 649)
    Oral Session Title: Gene Targeting and Gene Correction III
    Date and Time: Friday, May 12, 2017, 5.45 p.m. - 7.45 p.m.
    Location: Exhibit Hall A & B South