机译:Sample Preparation Matters for Peptide Mapping to Evaluate Deamidation of Adeno-Associated Virus Capsid Proteins Using Liquid Chromatography-Tandem Mass Spectrometry
机译:Adeno-Associated Virus-Mediated Gene Therapy for Patients' Fibroblasts, Induced Pluripotent Stem Cells, and a Mouse Model of Congenital Adrenal Hyperplasia
机译:Correction to: Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy, by Li et al. Hum Gene Ther 2020;31(15-16): 839-851. DOI: 10.1089/hum.2020.053
机译:Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting Dystrophia Myotonica Protein Kinase Transcripts for the Treatment of Myotonic Dystrophy Type 1