Gene Therapy and Oligonucleotide Compendium - Volume 1

Advanced Therapeutic Medicinal Products: Adeno-associated Virus (AAV) The Promise of Viral Vector Gene Therapy using AAV 7000 Monogenic Diseases 20 of 22 4 Years Children with spinal muscular atrophy (SMA) given the ability to thrive with Zolgensma® (AAV) at 91%…

Genome Sizing Methodology for Genome Integrity Analysis of Adeno-Associated Viruses (AAV) Jane Luo, Tingting Li, Fang Wang, Sahana Mollah, Handy Yowanto SCIEX, Brea, California Introduction Adeno-associated virus (AAV) is a small (25-nm) virus that is composed of a non-enveloped icosahedral…

Sensitive AAV Capsid Protein Impurity Analysis by CE Using Easy to Label Fluorescent Chromeo Dye P503 Marcia Santos; SCIEX, Brea, California Introduction AAV or Adeno associated virus (Figure 1) is a popular class of gene therapy delivery vehicle used mostly…

Accurate Analysis of Double Stranded DNA over an Extended Size Range Capillary Electrophoresis-Laser Induced Fluorescence Detection (CE-LIF) Jane Luo, Mukesh Malik, Marcia Santos, Sahana Mollah and Handy Yowanto SCIEX, Brea, California Introduction Analysis of double stranded DNA (ds DNA) is…