Journal: Clinical and Translational Medicine
Article Title: Muscle‐specific gene editing improves molecular and phenotypic defects in a mouse model of myotonic dystrophy type 1
doi: 10.1002/ctm2.70227
Figure Lengend Snippet: Improvement of bone health and body composition parameters in MyoAAV–C3/384‐treated homozygous DMSXL mice. A group of DMSXL male and female homozygous mice at age P5 received a systemic injection of MyoAAV vectors carrying Cas9 and sgC3/384 pair ( n = 7), and underwent whole‐body DEXA analysis at 16 weeks of age. Control animals were wild‐types (WT, n = 16) and untreated DMSXL homozygous mice (NT, n = 14) of both sexes. All data are represented as mean ± SEM and individual data points; data from male (open symbols) and female (filled symbols) mice are pooled. (A–C) Bone health parameters: bone mineral density (BMD) (A), bone mineral content (BMC) (B) and bone area (C). (A) BMD: MyoAAV–C3/384 vs. NT, unpaired t ‐test with Welch's correction, t = 2.595, df = 14, * p = .02. (B) BMC: MyoAAV–C3/384 vs. NT, unpaired t ‐test with Welch's correction, t = 3.371, df = 15, ** p = .004. (C) Bone area, WT vs. NT, Mann–Whitney test, U = 53, * p = .013; MyoAAV–C3/384 vs. NT, unpaired t ‐test with Welch's correction, t = 4.25, df = 16, *** p = .0006. (D–F) Body composition parameters: fat tissue percentage (D), lean tissue percentage (E) and fat/lean mass ratio (adiposity index, F). (D) Fat tissue %: WT vs. NT, Mann–Whitney test, U = 46, ** p = .0052; MyoAAV–C3/384 vs. NT, unpaired t ‐test with Welch's correction, t = 2.684, df = 17.14, * p = .015. (E) Lean tissue %: WT vs. NT, Mann–Whitney test, U = 45, ** p = .0045; MyoAAV–C3/384 vs. NT, unpaired t ‐test with Welch's correction, t = 2.684, df = 17.14, * p = .015. (F) Adiposity index, WT vs. NT, unpaired t ‐test with Welch's correction, t = 3.265, df = 15.37, ** p = .0051; MyoAAV–C3/384 vs. NT, unpaired t ‐test with Welch's correction, t = 2.643, df = 16.41, * p = .017.
Article Snippet: Bone health and body composition were comprehensively evaluated at the age of 4 months using Dual Energy X‐ray Absorptiometry (DEXA) analyser (The PARAMETER™ XPERT® 40 Cabinet X‐ray System, T00261_A 9.2019; Kubtec Scientific, 111 Research Drive Stratford, CT 06615, USA).
Techniques: Injection, Control, MANN-WHITNEY