Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy.

Autor: Chappell ME; Cell and Molecular Biology Affinity Group, University of Pennsylvania, Philadelphia, PA., Breda L; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA., Tricoli L; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA., Guerra A; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA., Jarocha D; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA.; Center for Cellular Immunotherapeutics, Translational and Correlative Studies Laboratory, University of Pennsylvania, Philadelphia, PA., Castruccio Castracani C; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA., Papp TE; Division of Infectious Diseases, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Tanaka N; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA., Hamilton N; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA., Triebwasser MP; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA.; Department of Pediatrics, University of Michigan, Ann Arbor, MI., Ghiaccio V; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA., Fedorky MT; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA., Gollomp KL; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA., Bochenek V; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA., Roche AM; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Everett JK; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Cook EJ; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Bushman FD; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Teawtrakul N; Division of Hematology, Department of Internal Medicine, Srinagarind Hospital, Khon Kaen University, Khon Kaen, Thailand., Glentis S; First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece., Kattamis A; First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece., Mui BL; Acuitas Therapeutics, Vancouver, BC, Canada., Tam YK; Acuitas Therapeutics, Vancouver, BC, Canada., Weissman D; Division of Infectious Diseases, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Abdulmalik O; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA., Parhiz H; Division of Infectious Diseases, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Rivella S; Cell and Molecular Biology Affinity Group, University of Pennsylvania, Philadelphia, PA.; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA.; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA.; Penn Center for Musculoskeletal Disorders, The Children's Hospital of Philadelphia, Philadelphia, PA.; Penn Institute for RNA Innovation, University of Pennsylvania, Philadelphia, PA.; Penn Institute for Regenerative Medicine, University of Pennsylvania, Philadelphia, PA.

Publikováno v: Blood [Blood] 2024 Oct 10; Vol. 144 (15), pp. 1633-1645.

Proliferative retinopathy and retinal detachment in pediatric atypical hemolytic uremic syndrome.

Autor: Tauqeer Z; Scheie Eye Institute, University of Pennsylvania, Philadelphia; Division of Pediatric Ophthalmology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania., Gollomp KL; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania., Bracha P; Gundersen Eye Institute, Gundersen Health System, La Crosse, Wisconsin., Maguire AM; Scheie Eye Institute, University of Pennsylvania, Philadelphia; Division of Pediatric Ophthalmology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. Electronic address: amaguire@pennmedicine.upenn.edu.

Publikováno v: Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus [J AAPOS] 2022 Feb; Vol. 26 (1), pp. 31-34. Date of Electronic Publication: 2021 Nov 14.