Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne’s Muscular Dystrophy

Angela Lek, Brenda Wong, Allison M. Keeler, +22

New England Journal of Medicine

Degenerative lumbar scoliosis in Chinese Han population: prevalence and relationship to age, gender, bone mineral density, and body mass index

Leilei Xu, Xu Sun, Shushu Huang, +6

European Spine Journal

Lumbar SpineBody CompositionDegenerative SpineDegenerative Lumbar ScoliosisBone Mineral Density +6

Applying genome-wide CRISPR-Cas9 screens for therapeutic discovery in facioscapulohumeral muscular dystrophy

Angela Lek, Keryn G. Woodman, Shushu Huang, +13

Science Translational Medicine

A three-year follow-up study evaluating clinical utility of exome sequencing and diagnostic potential of reanalysis

Jasmine Lee Fong Fung, Mullin H.C. Yu, Shushu Huang, +9

npj Genomic Medicine

Relationship between sagittal spinal alignment and the incidence of vertebral fracture in menopausal women with osteoporosis: a multicenter longitudinal follow-up study

Jian Dai, Xiaojuan Yu, Shushu Huang, +4

European Spine Journal

Bone DiseaseVertebral FractureLumbar SpineGynecologySagittal Spinal Alignment +7

Unexpected Death of a Duchenne Muscular Dystrophy Patient in an N-of-1 Trial of rAAV9-delivered CRISPR-transactivator

Angela Lek, Brenda Wong, Allison M. Keeler, +22

Custom TherapyIn Vivo Gene TherapyGeneticsImmunologyPathology +18

Exome sequencing in paediatric patients with movement disorders

Anna Ka‐Yee Kwong, Mandy Ho‐Yin Tsang, Jasmine Lee‐Fong Fung, +19

Orphanet Journal of Rare Diseases

Investigation of the 53 Markers in a DNA-Based Prognostic Test Revealing New Predisposition Genes for Adolescent Idiopathic Scoliosis

Leilei Xu, Shushu Huang, Xiaodong Qin, +5

Spine

Saturation mutagenesis-reinforced functional assays for disease-related genes

Kaiyue Ma, Shushu Huang, Kenneth Ng, +13

Cell

Disease-related GenesGeneticsGenetic EngineeringGene CharacterizationGenome Engineering +3