[HTML][HTML] The NIH somatic cell genome editing program

…, KS Lam, KW Leong, JE Levine, JA Lewis, CM Lutz… - Nature, 2021 - nature.com
The move from reading to writing the human genome offers new opportunities to improve
human health. The United States National Institutes of Health (NIH) Somatic Cell Genome …

[HTML][HTML] Absence epilepsy in tottering mutant mice is associated with calcium channel defects

CF Fletcher, CM Lutz, TN O'Sullivan, JD Shaughnessy… - Cell, 1996 - cell.com
… The closest proximal marker to tg, D8Mit78, was defined in the intercross, where it was
predicted to lie 0.04 ± 0.05 cM proximal to tgla (Figure 1). Only the Lyl1 locus remained …

C9orf72 is required for proper macrophage and microglial function in mice

…, CA Dangler, DM Underhill, HS Goodridge, CM Lutz… - Science, 2016 - science.org
Expansions of a hexanucleotide repeat (GGGGCC) in the noncoding region of the C9orf72
gene are the most common genetic cause of amyotrophic lateral sclerosis (ALS) and …

[PDF][PDF] C9orf72 BAC transgenic mice display typical pathologic features of ALS/FTD

…, DW Dickson, MB Harms, L Petrucelli, EB Lee, CM Lutz… - Neuron, 2015 - cell.com
… strength meter was positioned vertically, with the triangular metal transducer situated 40
cm … individually into the center of the arena and infrared beams record distance traveled (cm), …

[HTML][HTML] Sodium/hydrogen exchanger gene defect in slow-wave epilepsy mutant mice

GA Cox, CM Lutz, CL Yang, D Biemesderfer… - Cell, 1997 - cell.com
… A one cM resolution map was then constructed (Figure 3A), allowing the exclusion of several
candidate genes, including those encoding two serotonin receptors and neural syndecan-3…

Survival motor neuron protein in motor neurons determines synaptic integrity in spinal muscular atrophy

…, J Wooley, DJ Goldhamer, CM Lutz… - Journal of …, 2012 - Soc Neuroscience
… The total time upright, numbers of 5.5 × 5.5 cm grid squares crossed, and number of … SMN
expression and marked amelioration of the SMA phenotype (Lutz et al., 2011). After crossing …

Promoting validation and cross-phylogenetic integration in model organism research

…, AY Lin, KCK Lloyd, CM Lutz… - Disease models & …, 2022 - journals.biologists.com
… to histotomography for centimeter-size samples at submicron voxel resolutions (Yakovlev
et al., 2021) will be particularly useful for studying samples of millimeter to centimeter scale. …

[PDF][PDF] A platform for experimental precision medicine: The extended BXD mouse family

…, P Prins, MK Mulligan, S Roy, EG Williams, CM Lutz… - Cell systems, 2021 - cell.com
The challenge of precision medicine is to model complex interactions among DNA variants,
phenotypes, development, environments, and treatments. We address this challenge by …

[HTML][HTML] Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy

CM Lutz, S Kariya, S Patruni… - The Journal of …, 2011 - Am Soc Clin Investig
… This study was supported by SMA Foundation grants to CM Lutz, L. Pellizzoni, CE Henderson,
UR Monani, and Regeneron Pharmaceuticals. Additionally, UR Monani was supported by …

Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA

…, M Petrillo, KJ Swoboda, C Davis, CM Lutz… - Science translational …, 2021 - science.org
Gene replacement and pre-mRNA splicing modifier therapies represent breakthrough gene
targeting treatments for the neuromuscular disease spinal muscular atrophy (SMA), but …