Paper Review Records
All Paper Review Records
10 valid samples · Newest publication first
Review days = acceptance date − received date. PDF, DOI, and publisher-page sources are retained.
Gene augmentation therapy successfully treats mice with complete congenital stationary night blindness (cCSNB), improving retinal function and visual acuity
AuthorsNazarul Hasan; Cecilia A. Attaway; Mattia Di Paolo; Maureen A. McCall; Ronald G. Gregg
Affiliations1. Departments of Biochemistry & Molecular Genetics, University of Louisville, Louisville, USA; 2. Ophthalmology & Visual Science, University of Louisville, Louisville, USA; 3. Anatomical Sciences & Neurobiology, University of Louisville, Louisville, USA
Clinical response to systemic AAV gene therapy in a large animal model of late-stage lysosomal storage disease
AuthorsJacqueline E. Hunter; Caitlyn M. Molony; Dana L. Clarke; Wojciech Panek; Charles H. Vite; Sanjeev Chawla; Harish Poptani; John H. Wolfe
Affiliations1. Research Institute of Children’s Hospital of Philadelphia, Philadelphia, USA; 2. W.F. Goodman Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, USA; 3. Department of Small Animal Clinical Sciences, College of Veterinary Medicine, University of Florida, Gainesville, USA; 4. Department of Radiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, USA; 5. Department of Molecular and Clinical Cancer Medicine, University of Liverpool, Liverpool, UK; 6. Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, USA
Antimicrobial resistance and gene therapy: emerging molecular strategies for a global health threat
AuthorsA. Vitiello; M. Boccellino; A. Zovi; M. Bassetti
Affiliations1. Department of Health Prevention, Research and Emergencies, Italian Ministry of Health, Rome, Italy; 2. Università Link Campus di Roma, Rome, Italy; 3. Department of One health, italian Ministry of health, Roma, Italy; 4. University of Genova, Clinical Infectious Diseases Unit, San Martino Polyclinic Hospital-IRCCS, Genoa, Italy
The poly-(HA-GMA) hydrogel carrying AAV8-sTβRII alleviates scar formation in mice skin wound healing by inhibiting fibrosis
AuthorsJinhao Chen; Lijun Zhan; Jinyan Duan; Tianning Wang; Zenan Meng; Qianru Wang; Jianlin Yang; Xiaofei Huang; Yue Liao; Xinyu Song; Chunyu Cao
Affiliations1. Hubei Provincial Key Laboratory of Tumor Microenvironment and Immunotherapy, College of Basic Medical Sciences, China Three Gorges University, Yichang, China; 2. Affiliated Renhe Hospital of China Three Gorges University, Yichang, China; 3. Clinical Medical Research Center for Precision Diagnosis and Treatment of Lung Cancer and Management of Advanced Cancer Pain of Hubei Province, the First College of Clinical Medical Science, China Three Gorges University, Yichang, China
First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 study
AuthorsJirair K. Bedoyan; Thomas Morgan; Angela Sun; Hong Li; Daniel Gruskin; Marie Payton; Frederic Chereau; Eugene Scott Swenson; Qun Lin; Mark A. Kay; Jerry Vockley
Affiliations1. UPMC Children’s Hospital of Pittsburgh, Pittsburgh, USA; 2. University of Pittsburgh School of Medicine, Pittsburgh, USA; 3. Vanderbilt University School of Medicine, Nashville, USA; 4. Seattle Children’s Hospital, Seattle, USA; 5. Emory University, Atlanta, USA; 6. LogicBio Therapeutics, Lexington, USA; 7. Alexion, AstraZeneca Rare Disease, Boston, USA; 8. Stanford University, Stanford, USA
Building a gene editing lexicon: a model for rare and inherited disorders
AuthorsLeonard A. Valentino; Cedric Hermans; Donna Coffin; Wolfgang Miesbach; Maria Elisa Mancuso; Carmen Unzu; Micheala Jones; David E. Gutstein; William McKeown; Craig M. Kessler
Affiliations1. World Federation of Hemophilia USA, Albany, USA; 2. Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, USA; 3. Cliniques universitaires Saint-Luc, Université catholique de Louvain (UCLouvain), Brussels, Belgium; 4. Department of Data and Research, World Federation of Hemophilia, Montréal, Canada; 5. Department of Haemostaseology, University Hospital Frankfurt, Frankfurt, Germany; 6. Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Rozzano, Italy; 7. Humanitas University, Pieve Emanuele, Italy; 8. DNA&RNA Medicine Division, CIMA-Universidad de Navarra, Pamplona, Spain; 9. Regeneron Pharmaceuticals, Inc., Tarrytown, USA; 10. Care of Elderly Medicine, Antrim Area Hospital, Antrim, UK; 11. Hemophilia and Thrombophilia Comprehensive Treatment Center, Georgetown University Medical Center, Washington, USA
Topical application of Cas9 ribonucleoproteins inhibits corneal neovascularization in a mouse model of alkali burn injury
AuthorsSeok Jae Lee; Bae-Geun Nam; Sung-Ah Hong; Dong Hyun Jo; Sang-Mok Lee; Sangsu Bae; Jeong Hun Kim
Affiliations1. Fight against Angiogenesis-Related Blindness (FARB) Laboratory, Clinical Research Institute, Seoul National University Hospital, Seoul, Republic of Korea; 2. Global Excellence Center for Gene & Cell Therapy (GEC-GCT), Seoul National University Hospital, Seoul, Republic of Korea; 3. Department of Ophthalmology, Seoul National University Hospital, Seoul, Republic of Korea; 4. Genomic Medicine Institute, Seoul National University College of Medicine, Seoul, Republic of Korea; 5. Divison of Bio Research, SK Bioscience, Seongnam-si, Republic of Korea; 6. Department of Anatomy & Cell Biology, Seoul National University College of Medicine, Seoul, Republic of Korea; 7. Department of Cornea, External Disease & Refractive Surgery, HanGil Eye Hospital, Incheon, Republic of Korea; 8. Department of Ophthalmology, Catholic Kwandong University College of Medicine, Gangneung-si, Republic of Korea; 9. Department of Biomedical Sciences, Seoul National University College of Medicine, Seoul, Republic of Korea; 10. Institute of Reproductive Medicine and Population, Seoul National University College of Medicine, Seoul, Republic of Korea
Spatial transcriptomics and single-nucleus RNA sequencing reveal rAAV2- and rAAV9-specific transduction signatures in the mouse liver
AuthorsBettina Amberg; Fabian Köchl; Nadine Kumpesa; Megana Prasad; Filip Bochner; Mar Hernández-Obiols; Michael B. Otteneder; Lucas Stalder; Frances Shaffo; Ali Nowrouzi; David Markusic; Hélène Haegel; Rebecca Xicluna; Marc Sultan; Björn Jacobsen; Sven Rottenberg; Alberto Valdeolivas; Petra C. Schwalie; Kerstin Hahn
Affiliations1. Roche Pharma Research and Early Development, Pharmaceutical Sciences, Roche Innovation Center Basel, Basel, Switzerland; 2. Institute of Animal Pathology, Vetsuisse Faculty, University of Bern, Bern, Switzerland; 3. Department of Biosystems Science and Engineering, Pharmaceutical Sciences, ETH Zürich, Basel, Switzerland; 4. Spark Therapeutics Inc., Philadelphia, USA; 5. Bern Center for Precision Medicine, Department for BioMedical Research, University of Bern, Bern, Switzerland
AAV-mediated gene replacement therapy for LRAT -associated retinitis pigmentosa: a proof-of-concept study in a patient-based rat model
AuthorsA. M. El-Kalaani; J. B. Ten Brink; C. J. F. Boon; C. Koster
Affiliations1. Department of Ophthalmology, Amsterdam UMC, University of Amsterdam (UvA), Amsterdam, The Netherlands; 2. Department of Human Genetics, Amsterdam UMC, University of Amsterdam (UvA), Amsterdam, The Netherlands; 3. Department of Ophthalmology, Leiden University Medical Center (LUMC), Leiden, The Netherlands; 4. Emma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, The Netherlands
In vitro and in vivo rescue of dopaminergic neurons in Parkinson’s disease models after Parkin gene therapy
AuthorsTakeshi Hioki; Masaaki Nishimura; Xiuxia Sun; Sarah Melissa Jacobo; Miyu Nakayama; Mitsuhiro Nishihara; Kimio Tohyama; Po-Ting Liu; Takuro Okai; Maiko Tanaka; Gabriele Proetzel
Affiliations1. Research, Takeda Pharmaceutical Company Limited, Fujisawa, Japan; 2. Research, Takeda Development Center Americas Inc, Cambridge, USA
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