Identification of a gene, ABCG5, important in the regulation of dietary cholesterol absorption

Mi‐Hye Lee, Kangmo Lu, Star Hazard, +11

Nature Genetics

OxysterolBiochemistryLipid NutritionNatural SciencesGenetics +6

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

Yang Yang, Lili Wang, Peter Bell, +9

Nature Biotechnology

Systems BiologyHepatologyCas9-mediated CorrectionLiver PhysiologyInherited Metabolic Disease +8

7-Dehydrocholesterol–dependent proteolysis of HMG-CoA reductase suppresses sterol biosynthesis in a mouse model of Smith-Lemli-Opitz/RSH syndrome

Barbara U. Fitzky, Fabian F. Moebius, Hitoshi Asaoka, +16

Journal of Clinical Investigation

7-Dehydrocholesterol–dependent proteolysis of HMG-CoA reductase suppresses sterol biosynthesis in a mouse model of Smith-Lemli-Opitz/RSH syndrome

Barbara U. Fitzky, Fabian F. Moebius, Hitoshi Asaoka, +16

Journal of Clinical Investigation

A mouse model of sitosterolemia: absence of Abcg8/sterolin-2 results in failure to secrete biliary cholesterol

Eric L. Klett, Kangmo Lu, Astrid Kosters, +14

BMC Medicine

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals

Christian Hinderer, Peter Bell, Nathan Katz, +12

Human Gene Therapy

ImmunologyTransduction EfficiencyGene DeliveryBiomedical EngineeringIntrathecal Routes +17

Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna

Christian Hinderer, Peter Bell, Charles H. Vite, +7

Molecular Therapy — Methods & Clinical Development

Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids

Lili Wang, Peter Bell, Suryanarayan Somanathan, +7

Molecular Therapy

Direct injection into the dorsal root ganglion: Technical, behavioral, and histological observations

Gregory Fischer, Sandra Kostić, Hiroyuki Nakai, +4

Journal of Neuroscience Methods

Ganglion CellPeripheral Nerve InjuryNeurophysiologyNeuroanatomyHistological Observations +10

The use of the Dhcr7 knockout mouse to accurately determine the origin of fetal sterols

G S Tint, Hongwei Yu, Quan Shang, +2

Journal of Lipid Research