Spotlight on Gene Therapy: finding a Cure

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.”

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