WT‐HSCT, LV. IDS, and LV. IDS

Slides:

Advertisements

Similar presentations

Hyp mice show hypertension and increased NCC expression and phosphorylation A–ESerum intact Fgf23 concentration (A, n = 8–9, Student's t‐test, *P =

Advertisements

Impact of triple combination of gemcitabine‐based chemoirradiation with anti‐PD‐L1 in the KPC tumor allograft Impact of triple combination of gemcitabine‐based.

Morphology of white adipose tissue and muscle in S1/3 KO mice of different ages Morphology of white adipose tissue and muscle in S1/3 KO mice of different.

Loss of Hdac3 impairs Ar signaling in mouse prostate tissues and has no effect on apoptosis Loss of Hdac3 impairs Ar signaling in mouse prostate tissues.

Metabolic effects of TUG‐891 are reduced or absent in GPR120‐deficient mice Metabolic effects of TUG‐891 are reduced or absent in GPR120‐deficient mice.

Morphological analyses of peripheral blood and bone marrow cells isolated from leukemic mice. Morphological analyses of peripheral blood and bone marrow.

PERK activator reduces behavioral deficits and neurodegeneration in vivo PERK activator reduces behavioral deficits and neurodegeneration in vivo A–CYoung.

Neuro‐inflammatory cytokines, brain microglial activation, and working memory deficits are normalized using LV.IDS.ApoEII, but not LV.IDS in MPS II mice.

Treatment with p38γ/p38δ inhibitor shows antifungal effects in vivo

[68Ga]Pentixafor PET imaging of MM xenografts Real‐time PCR analysis of cxcr4 transcript expression levels in HeLa (negative control) and in MM cell lines.

TFE3 regulates glucose homeostasis

AAV8‐hAAT‐FGF21 treatment improves liver fibrosis

Differential accumulation of substrates in vivo

Treatment with FGF21‐encoding vectors reverses hepatic steatosis and fibrosis Treatment with FGF21‐encoding vectors reverses hepatic steatosis and fibrosis.

Dual LGR5 and KI67 knock‐in PDOs enable separation of quiescent and cycling LGR5+ CRC cells Dual LGR5 and KI67 knock‐in PDOs enable separation of quiescent.

Metabolic profile of Tfe3 KO mice

Both haematopoietic and non‐haematopoietic compartments are involved in myocarditis development.A.Kaplan‐Meier survival curves showing survival of bone.

Preceding knockdown of FoxO3 reduces inhibitory effect of UCN‐01 on human lung fibroblast proliferation and myofibroblast transdifferentiation in response.

Impaired fear extinction precedes memory decline in Fmn2 mutant mice

ADAM8 promotes cancer cell adhesion to endothelial cells via β1‐integrin activation ABlood was drawn using the submandibular collection method from 4 mice/group.

Effects of GM1 on astroglial and microglial markers

Stat3 promotes lung fibrosis and collagen synthesis independent of Smad3.A.Masson's trichrome stained sections of lung from gp130wt (wt), Smad3−/− (Smad3−/−),

TFE3 prevents diet‐induced obesity and metabolic syndrome

HSPC and T‐cell transduction

MI during active phase (ZT13) leads to infarct expansion and reduced cardiac function MI during active phase (ZT13) leads to infarct expansion and reduced.

Expression of PD‐1 on PBMC and malignant exudate T cells

Stat3C/C mice are more prone to Th17 differentiation. A

PERK activator treatment protects neurite network

Neuro‐inflammatory cytokines, brain microglial activation, and working memory deficits are normalized using LV.IDS.ApoEII, but not LV.IDS in MPS II mice.

Treatment with FGF21‐encoding vectors reverses hepatic steatosis and fibrosis Treatment with FGF21‐encoding vectors reverses hepatic steatosis and fibrosis.

Intra‐eWAT administration of FGF21 vectors in ob/ob mice

Nec‐1 inhibits the phosphorylation and aggregation of tau

High frequency and unique tumor‐initiating cells (TICs) in Pten/p53‐deficient mice Flow cytometry profiles of indicated tumors with the CD24 and CD49f.

Hdac3 deletion decreases AKT phosphorylation and tumor growth in Pten knockout prostate cancer Hdac3 deletion decreases AKT phosphorylation and tumor growth.

Impaired ECM protein synthesis in infarcted area of S100a4cre+/− × Fstl1flox/flox mice Impaired ECM protein synthesis in infarcted area of S100a4cre+/−

Treatment with AAV8‐hAAT‐FGF21 improves insulin sensitivity and glucose tolerance Treatment with AAV8‐hAAT‐FGF21 improves insulin sensitivity and glucose.

Mohamed A. Saleh et al. BTS 2016;1:

TGF‐β1 treatment induces the chromatin binding of Smad2/3/4 in 4T1 cells TGF‐β1 treatment induces the chromatin binding of Smad2/3/4 in 4T1 cells A, BWestern.

Normal priming and expansion of functional T cells in the spleen during concurrent co‐infection Normal priming and expansion of functional T cells in the.

IL‐23‐induced psoriasis‐like skin disease is ameliorated in IL‐23−/− mice IL‐23‐induced psoriasis‐like skin disease is ameliorated in IL‐23−/− mice ARepresentative.

Assessment of intracerebral micro‐hemorrhages in APP23‐transgenic mice, dosed orally with CNP520 at 30 mg/kg for 3 months Assessment of intracerebral micro‐hemorrhages.

LV.IDS and LV.IDS.ApoEII improve brain‐specific IDS activity and express supra‐physiological levels of active IDS in peripheral organs LV.IDS and LV.IDS.ApoEII.

mGPDH is not essential to muscle development

Impact of ONX 0914 on abundance and dissemination of monocytes/macrophages during CVB3 infection Impact of ONX 0914 on abundance and dissemination of monocytes/macrophages.

High‐fat diet augments gut permeability, metabolic endotoxemia, and systemic inflammation High‐fat diet augments gut permeability, metabolic endotoxemia,

DOK7 gene therapy suppresses motor nerve terminal degeneration at the NMJ in ALS mice DOK7 gene therapy suppresses motor nerve terminal degeneration at.

NOD2 deletion exacerbates diet‐induced bacterial translocation, inflammation, and impairs insulin action in the liver A–CQuantification of bacterial DNA.

MiR‐34a‐5p functionally contributes to arrested lung alveolarization in response to hyperoxia miR‐34a‐5p functionally contributes to arrested lung alveolarization.

Therapeutic role of TR in HFD‐established diabetic mice

The CHCH domain is necessary for mitochondrial import of CHCHD10

DHEA shortens circadian period

P2X4R blockade increases autoimmune inflammation

T22‐GFP‐H6‐FdU‐induced depletion of CXCR4‐overexpressing cancer cells in tumor tissue T22‐GFP‐H6‐FdU‐induced depletion of CXCR4‐overexpressing cancer cells.

UCN‐01 inhibits FoxO3 inactivation in human lung fibroblasts stimulated with growth factors UCN‐01 inhibits FoxO3 inactivation in human lung fibroblasts.

Therapeutic activity of gal‐encapsulated cytotoxic drugs on tumor xenografts Therapeutic activity of gal‐encapsulated cytotoxic drugs on tumor xenografts.

ATAD1 physically interacts with the TA protein, GOS28, and is required to limit the level of mislocalized GOS28 on mitochondria in mammals Human dermal.

Tissue‐specific differences in the levels of CoQ9, CoQ10, and DMQ9, and in the DMQ9/CoQ9 ratio after β‐RA treatment in Coq9R239X mice Tissue‐specific differences.

Favorable safety profile of LV‐mediated intracerebral gene therapy

Antagonizing miR‐34a function partially restores proper lung alveolarization Antagonizing miR‐34a function partially restores proper lung alveolarization.

Impact of ONX 0914‐induced neutrophilia during CVB3 infection

Catalytic activity of mTERT is needed for increased healthspan and life‐span extension in AAV9‐mTERT treated mice.A.Scheme of mTERT‐wt and mTERT‐DN, a.

GM1 decreases neuropathology and weight loss in R6/2 mice

P2X4R blockade increases pro‐inflammatory gene expression after EAE

Nilotinib inhibits DDR1 invasive activity of patient‐derived CRC cells

Symptomatic rescue of double‐transgenic mice without decreased protein aggregation The expression of exogenous HMGB1‐FLAG (detected with an anti‐FLAG antibody)

Supplementary Figure 5: Effect of S48168 on normalized organ and skeletal muscle weights after 12 weeks of treatment for all the experimental groups from.

Limiting neutrophilic inflammation during active phase (ZT13) reduces MI damage Limiting neutrophilic inflammation during active phase (ZT13) reduces MI.

KlbHET mice exhibit altered estrous cycle and subfertility

SEMA3C regulates prostate cancer cell growth

Niacin suppresses pro‐inflammatory cytokine expression in macrophages in DSS‐induced colitis in mice Niacin suppresses pro‐inflammatory cytokine expression.

Presentation transcript:

WT‐HSCT, LV. IDS, and LV. IDS WT‐HSCT, LV.IDS, and LV.IDS.ApoEII normalize skeletal defects, peripheral inflammation, and heart failure markers in MPS II mice WT‐HSCT, LV.IDS, and LV.IDS.ApoEII normalize skeletal defects, peripheral inflammation, and heart failure markers in MPS II mice ARepresentative X‐ray images of control and treated MPS II mouse craniums at 8 months of age. White arrows indicate the area where zygomatic arches are measured.B–DZygomatic arch widths (B), humerus widths (C), and femur widths (D) were analyzed using ImageJ software, n = 5–11.ERepresentation of the accelerating rotarod.FEight‐month‐old control and treated mice were trialed three times on the accelerating rotarod for a maximum of 300 s (4–4 rpm over 300 s), n = 12–16 mice/group.G–ICytokine bead arrays measuring MCP‐1 (G), MIP‐1α (H), and RANTES (I) were performed on liver lysates of 8‐month‐old mice using flow cytometry, n = 4–6.J, KGene expression of Nppb (J) and Myh7 (K) in hearts of control and treated MPS II animals (WT n = 3; MPS II n = 4; WT‐HSCT, LV.IDS, and LV.IDS.ApoEII n = 6).LIDS‐specific IgG antibody titers were measured in plasma (pos. control n = 1, neg. control (uninjected MPS II plasma) n = 6, LV.IDS/LV.IDS.ApoEII n = 6).Data information: Data are mean ± SEM, one‐way ANOVA, *P < 0.05, **P < 0.01, ***P < 0.001, ****P < 0.0001 vs. MPS II. Hélène FE Gleitz et al. EMBO Mol Med. 2018;emmm.201708730 © as stated in the article, figure or figure legend