A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells The authors describe a CRISPR/Cas9 platform applicable to 60% of Duchenne muscular dystrophy (DMD) patient mutations. Internally deleted dystrophin was functional as demonstrated by improved membrane integrity and restoration of the dystrophin glycoprotein complex in vitro and in vivo. [Cell Stem Cell] Abstract | Graphical Abstract | Press Release Liraglutide Compromises Pancreatic β Cell Function in a Humanized Mouse Model Researchers showed that prolonged daily liraglutide treatment of >200 days in humanized mice, transplanted with human pancreatic islets in the anterior chamber of the eye, is associated with compromised release of human insulin and deranged overall glucose homeostasis. [Cell Metab] Abstract | Graphical Abstract | Press Release Endothelial Dicer Promotes Atherosclerosis and Vascular Inflammation by miRNA-103-Mediated Suppression of KLF4 Investigators showed that reduced endothelial expression of the RNAse Dicer, which generates almost all mature miRNAs, decreases monocyte adhesion, endothelial C–X–C motif chemokine 1 expression, atherosclerosis and the lesional macrophage content in apolipoprotein E knockout mice after exposure to a high-fat diet. [Nat Commun] Full Article | Press Release Promoting Axon Regeneration in the Adult CNS by Modulation of the Melanopsin/GPCR Signaling Researchers found that subtypes of intrinsically photosensitive retinal ganglion cells (ipRGCs) in mice maintained high mammalian target of rapamycin (mTOR) levels after axotomy and that the light-sensitive G protein-coupled receptor (GPCR) melanopsin mediated this sustained expression. Melanopsin overexpression in the RGCs stimulated axonal regeneration after optic nerve crush by up-regulating mTOR complex 1. [Proc Natl Acad Sci USA] Abstract | Full Article | Press Release IGF-1 C Domain–Modified Hydrogel Enhances Cell Therapy for AKI Scientists synthesized a bioactive hydrogel by immobilizing the C domain peptide of IGF-1 (IGF-1C) on chitosan, and hypothesized that this hydrogel could provide a favorable niche for adipose-derived mesenchymal stem cells and thereby enhance cell survival in an AKI model. [J Am Soc Nephrol] Abstract AAV-Mediated Gene Therapy Halts Retinal Degeneration in PDE6β-Deficient Dogs Scientists provide a detailed assessment of the long-term effects of gene therapy on the spatiotemporal pattern of retinal degeneration in rod-cone dysplasia type 1 dogs treated at 20 days of age. Whereas no rods were observed outside the bleb or in untreated eyes, gene transfer halted rod degeneration in all vector-exposed regions. [Mol Ther] Abstract Basic FGF or VEGF Gene Therapy Corrects Insufficiency in the Intrinsic Healing Capacity of Tendons By basic fibroblast growth factor (bFGF) or vascular endothelial growth factor (VEGF) gene therapy via adeno-associated viral type-2 vector to produce supernormal amounts of bFGF or VEGF intrinsically in the tendon, researchers effectively corrected the insufficiency of the tendon healing capacity. [Sci Rep] Full Article 3D Printing Surgical Implants at the Clinic: A Experimental Study on Anterior Cruciate Ligament Reconstruction A well-defined, orthogonal, porous PLA screw-like scaffold was printed, then coated with hydroxyapatite to improve its osteoconductivity. As an internal fixation as well as an ideal cell delivery system, the osteogenic scaffold loaded with mesenchymal stem cells were evaluated through both in vitro and in vivo tests to observe bone-ligament healing via cell therapy. [Sci Rep] Full Article Transplantation of Neural Progenitor Cells in Chronic Spinal Cord Injury Investigators tested whether transplanted neural progenitor cells can also improve functional recovery after chronic spinal cord injury alone or in combination with the reduction of glial scar and neurotrophic support. [Neuroscience] Abstract Mifepristone-Inducible Transgene Expression in Neural Progenitor Cells In Vitro and In Vivo The authors used a lentivirus-based vector platform for the ex vivo production of mifepristone-inducible murine neural progenitor cells that express transgenes of interest. Inducible cells were then transplanted into the brains of rodents, where they exhibited appropriate mifepristone-inducible expression. [Gene Ther] Abstract Effects of FVIII Immunity on Hepatocyte and Hematopoietic Stem Cell–Directed Gene Therapy of Murine Hemophilia A As clinical immune responses to coagulation factors VIII (FVIII) and IX differ, scientists investigated the ability of liver-directed adeno-associated viral-FVIII gene therapy to affect FVIII immunity in hemophilia A mice. [Mol Ther Methods Clin Dev] Full Article |