Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies.

Autor: Chhabra A; Pfizer Inc., New York, NY, USA., Bashirians G; Pfizer Inc., Cambridge, MA, USA., Petropoulos CJ; Labcorp-Monogram Biosciences, South San Francisco, CA, USA., Wrin T; Labcorp-Monogram Biosciences, South San Francisco, CA, USA., Paliwal Y; Pfizer Inc., Collegeville, PA, USA., Henstock PV; Pfizer Inc., Andover, MA, USA., Somanathan S; Pfizer Inc., Cambridge, MA, USA., da Fonseca Pereira C; Pfizer Australia, Sydney, NSW, Australia., Winburn I; Pfizer Ltd., Walton on the Hill, Surrey, UK., Rasko JEJ; University of Sydney, Central Clinical School, Faculty of Medicine & Health, Sydney, NSW, Australia.; Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, Sydney, NSW, Australia.; Gene and Stem Cell Therapy Program, Centenary Institute University of Sydney, Sydney, NSW, Australia.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2024 May 29; Vol. 32 (3), pp. 101273. Date of Electronic Publication: 2024 May 29 (Print Publication: 2024).

Complement Activation by Adeno-Associated Virus-Neutralizing Antibody Complexes.

Autor: West C; Pfizer, Inc., Rare Disease Research Unit, Cambridge, Massachusetts, USA., Federspiel JD; Pfizer, Inc., Drug Safety Research & Development, Andover, Massachusetts, USA., Rogers K; Pfizer, Inc., Drug Safety Research & Development, Groton, Connecticut, USA., Khatri A; Pfizer, Inc., Rare Disease Research Unit, Cambridge, Massachusetts, USA., Rao-Dayton S; Pfizer, Inc., Rare Disease Research Unit, Kit Creek, North Carolina, USA., Ocana MF; Pfizer, Inc., Drug Safety Research & Development, Andover, Massachusetts, USA., Lim S; Pfizer, Inc., Biomedicine Design, Cambridge, Massachusetts, USA., D'Antona AM; Pfizer, Inc., Biomedicine Design, Cambridge, Massachusetts, USA., Casinghino S; Pfizer, Inc., Drug Safety Research & Development, Groton, Connecticut, USA., Somanathan S; Pfizer, Inc., Rare Disease Research Unit, Cambridge, Massachusetts, USA.

Publikováno v: Human gene therapy [Hum Gene Ther] 2023 Jun; Vol. 34 (11-12), pp. 554-566.

Automatic detection of foreign body objects in neurosurgery using a deep learning approach on intraoperative ultrasound images: From animal models to first in-human testing.

Autor: Abramson HG; Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, United States., Curry EJ; Department of Neurosurgery, Johns Hopkins University School of Medicine, Baltimore, MD, United States., Mess G; Department of Biomedical Engineering, Johns Hopkins University, Baltimore, MD, United States., Thombre R; Department of Biomedical Engineering, Johns Hopkins University, Baltimore, MD, United States., Kempski-Leadingham KM; Department of Neurosurgery, Johns Hopkins University School of Medicine, Baltimore, MD, United States., Mistry S; Department of Engineering Science, University of Toronto, Toronto, ON, Canada., Somanathan S; Department of Biomedical Engineering, Johns Hopkins University, Baltimore, MD, United States., Roy L; Roy Illustration, Des Moines, IA, United States., Abu-Bonsrah N; Department of Neurosurgery, Johns Hopkins University School of Medicine, Baltimore, MD, United States., Coles G; Applied Physics Lab, Johns Hopkins University, Laurel, MD, United States., Doloff JC; Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, United States.; Department of Materials Science and Engineering, Johns Hopkins University, Baltimore, MD, United States., Brem H; Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, United States.; Department of Neurosurgery, Johns Hopkins University School of Medicine, Baltimore, MD, United States., Theodore N; Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, United States.; Department of Neurosurgery, Johns Hopkins University School of Medicine, Baltimore, MD, United States., Huang J; Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, United States.; Department of Neurosurgery, Johns Hopkins University School of Medicine, Baltimore, MD, United States., Manbachi A; Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, United States.; Department of Neurosurgery, Johns Hopkins University School of Medicine, Baltimore, MD, United States.; Department of Electrical Engineering and Computer Science, Johns Hopkins University, Baltimore, MD, United States.; Department of Mechanical Engineering, Johns Hopkins University, Baltimore, MD, United States.; Department of Anesthesiology, Johns Hopkins University School of Medicine, Baltimore, MD, United States.

Publikováno v: Frontiers in surgery [Front Surg] 2022 Nov 30; Vol. 9, pp. 1040066. Date of Electronic Publication: 2022 Nov 30 (Print Publication: 2022).

In vivo overexpression of frataxin causes toxicity mediated by iron-sulfur cluster deficiency.

Autor: Huichalaf C; Rare Disease Research Unit, Worldwide Research, Development and Medical, Pfizer Inc., 610 Main Street, Cambridge, MA 02139, USA., Perfitt TL; Rare Disease Research Unit, Worldwide Research, Development and Medical, Pfizer Inc., 610 Main Street, Cambridge, MA 02139, USA., Kuperman A; Rare Disease Research Unit, Worldwide Research, Development and Medical, Pfizer Inc., 610 Main Street, Cambridge, MA 02139, USA., Gooch R; Rare Disease Research Unit, Worldwide Research, Development and Medical, Pfizer Inc., 610 Main Street, Cambridge, MA 02139, USA., Kovi RC; Drug Safety Research and Development, Worldwide Research, Development and Medical, Pfizer Inc., Cambridge, MA 02139, USA., Brenneman KA; Drug Safety Research and Development, Worldwide Research, Development and Medical, Pfizer Inc., Cambridge, MA 02139, USA., Chen X; Comparative Medicine, Worldwide Research, Development and Medical, Pfizer Inc., Cambridge, MA 02139, USA., Hirenallur-Shanthappa D; Comparative Medicine, Worldwide Research, Development and Medical, Pfizer Inc., Cambridge, MA 02139, USA., Ma T; Rare Disease Research Unit, Worldwide Research, Development and Medical, Pfizer Inc., 610 Main Street, Cambridge, MA 02139, USA., Assaf BT; Drug Safety Research and Development, Worldwide Research, Development and Medical, Pfizer Inc., Cambridge, MA 02139, USA., Pardo I; Drug Safety Research and Development, Worldwide Research, Development and Medical, Pfizer Inc., Cambridge, MA 02139, USA., Franks T; Drug Safety Research and Development, Worldwide Research, Development and Medical, Pfizer Inc., Cambridge, MA 02139, USA., Monarski L; Drug Safety Research and Development, Worldwide Research, Development and Medical, Pfizer Inc., Cambridge, MA 02139, USA., Cheng TW; Rare Disease Research Unit, Worldwide Research, Development and Medical, Pfizer Inc., 610 Main Street, Cambridge, MA 02139, USA., Le K; Rare Disease Research Unit, Worldwide Research, Development and Medical, Pfizer Inc., 610 Main Street, Cambridge, MA 02139, USA., Su C; Rare Disease Research Unit, Worldwide Research, Development and Medical, Pfizer Inc., 610 Main Street, Cambridge, MA 02139, USA., Somanathan S; Rare Disease Research Unit, Worldwide Research, Development and Medical, Pfizer Inc., 610 Main Street, Cambridge, MA 02139, USA., Whiteley LO; Drug Safety Research and Development, Worldwide Research, Development and Medical, Pfizer Inc., Cambridge, MA 02139, USA., Bulawa C; Rare Disease Research Unit, Worldwide Research, Development and Medical, Pfizer Inc., 610 Main Street, Cambridge, MA 02139, USA., Pregel MJ; Rare Disease Research Unit, Worldwide Research, Development and Medical, Pfizer Inc., 610 Main Street, Cambridge, MA 02139, USA., Martelli A; Rare Disease Research Unit, Worldwide Research, Development and Medical, Pfizer Inc., 610 Main Street, Cambridge, MA 02139, USA.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2022 Feb 07; Vol. 24, pp. 367-378. Date of Electronic Publication: 2022 Feb 07 (Print Publication: 2022).

Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia.

Autor: Wang L; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Muthuramu I; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Somanathan S; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Zhang H; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Bell P; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., He Z; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Yu H; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Zhu Y; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Tretiakova AP; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Wilson JM; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2021 May 05; Vol. 22, pp. 1-10. Date of Electronic Publication: 2021 May 05 (Print Publication: 2021).