In vivo gene editing via homology-independent targeted integration for adrenoleukodystrophy treatment
Sung-Ah Hong, Jung Hwa Seo, Soohyun Wi, Eul Sik Jung, Jihyeon Yu, Gue‐Ho Hwang, Ji Hea Yu, Ahreum Baek, Soeon Park, Sangsu Bae, Sung‐Rae Cho
Topics & Concepts
Genome editingHomology (biology)In vivoBiologyAdrenoleukodystrophyGeneSequence homologyComputational biologyGeneticsCRISPRBase sequencePeroxisomeRNA regulation and diseaseCRISPR and Genetic EngineeringVirus-based gene therapy research