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Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting

Autor: Juan A. Bueren, Susana Navarro, Jennifer E. Adair, Paula Río, Michael Antoniou, Cynthia C. Bartholomae, Julián Sevilla, Pamela S. Becker, Anne Galy, Adrian J. Thrasher, Bruce R. Blazar, David A. Williams, Marina Cavazzana-Calvo, Thomas Carroll, Luigi Naldini, Robert Dalgleish, Els Verhoeyen, Helmut Hanenberg, Raffaele Renella, H. Bobby Gaspar, D. Wade Clapp, Dirk Lindeman, Hans-Peter Kiem, Jakub Tolar, John E. Wagner, Manfred Schmidt, Christof von Kalle

Publikováno v: Molecular Therapy; Vol 19
Molecular Therapy
Molecular Therapy, Cell Press, 2011, 19 (7), pp.1193-8. ⟨10.1038/mt.2011.78⟩
Molecular Therapy, 2011, 19 (7), pp.1193-8. ⟨10.1038/mt.2011.78⟩

Survival rates after allogeneic hematopoietic cell transplantation (HCT) for Fanconi anemia (FA) have increased dramatically since 2000. However, the use of autologous stem cell gene therapy, whereby the patient's own blood stem cells are modified to

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::972e99d15287ee06abcb4d938647669c

SCHIZANGIELLA INFECTIONS IN AN EASTERN RATSNAKE ( PANTHEROPHIS ALLEGHANIENSIS ) AND A TIMBER RATTLESNAKE ( CROTALUS HORRIDUS ).

Autor: Velez ED; Metropolitan Veterinary Hospital Exotics Department, Norton, OH 44302, USA, emily.velez@nva.com., Garner MM; Northwest ZooPath, Monroe, WA 98272, USA., Jacobson ER; Department of Comparative, Diagnostic, and Population Medicine, College of Veterinary Medicine, University of Florida, Gainesville, FL 32610, USA., Badial P; Department of Comparative, Diagnostic, and Population Medicine, College of Veterinary Medicine, University of Florida, Gainesville, FL 32610, USA., Bonar CJ; Zoo New England, Boston, MA 02121, USA., Riggs GL; Metropolitan Veterinary Hospital Exotics Department, Norton, OH 44302, USA., Adair JE; Metropolitan Veterinary Hospital Exotics Department, Norton, OH 44302, USA., Wellehan JFX; Department of Comparative, Diagnostic, and Population Medicine, College of Veterinary Medicine, University of Florida, Gainesville, FL 32610, USA., Ossiboff RJ; Department of Comparative, Diagnostic, and Population Medicine, College of Veterinary Medicine, University of Florida, Gainesville, FL 32610, USA.

Publikováno v: Journal of zoo and wildlife medicine : official publication of the American Association of Zoo Veterinarians [J Zoo Wildl Med] 2024 Jun; Vol. 55 (2), pp. 540-546.

The translational gap for gene therapies in low- and middle-income countries.

Autor: Doxzen KW; Strategic Analysis Inc., Arlington, VA, USA., Adair JE; Translational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.; Departments of Medical Oncology and Laboratory Medicine and Pathology, University of Washington, Seattle, WA, USA., Fonseca Bazzo YM; Department of Pharmaceutical Sciences, Faculty of Health Sciences, University of Brasília, Brasília, DF, Brazil., Bukini D; Sickle Cell Programme, Department of Haematology and Blood Transfusion, Muhimbili University of Health & Allied Sciences (MUHAS), Dar-es-Salaam, Tanzania.; SickleInAfrica Clinical Coordinating Center, Muhimbili University of Health & Allied Sciences (MUHAS), Dar-es-Salaam, Tanzania., Cornetta K; Indiana University School of Medicine, Indianapolis, IN, USA., Dalal V; Department of Health Research, Ministry of Health and Family Welfare, Government of India, New Delhi, India., Guerino-Cunha RL; Centro Paulista de Oncologia, Grupo Oncoclínicas, São Paulo, SP, Brazil.; Instituto Oncoclínicas, Rio de Janeiro, RJ, Brazil., Hongeng S; Division of Hematology and Oncology, Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand., Jotwani G; Indian Council of Medical Research, New Delhi, India., Kityo-Mutuluuza C; Joint Clinical Research Centre (JCRC), Kampala, Uganda., Lakshmanan K; Department of Pediatric Hematology and Oncology, Yale School of Medicine, New Haven, CT, USA., Mahlangu J; Department of Molecular Medicine and Haematology, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa., Makani J; Sickle Cell Programme, Department of Haematology and Blood Transfusion, Muhimbili University of Health & Allied Sciences (MUHAS), Dar-es-Salaam, Tanzania.; SickleInAfrica Clinical Coordinating Center, Muhimbili University of Health & Allied Sciences (MUHAS), Dar-es-Salaam, Tanzania.; Centre for Haematology, Department of Immunology and Inflammation, Imperial College London, London, UK., Mathews V; Department of Haematology, Christian Medical College (CMC), Vellore, India., Ozelo MC; INCT Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, SP, Brazil., Rangarajan S; Advanced Center for Oncology, Haematology & Rare Disorders (ACOHRD), K. J. Somaiya Medical College & Research Center, Somaiya Ayurvihar, Mumbai, Maharashtra, India.; Faculty of Medicine, University of Southampton, Southampton, UK., Scholefield J; Bioengineering and Integrated Genomics Group, Next Generation Health Cluster, Council for Scientific and Industrial Research, Pretoria, South Africa.; Department of Human Biology, Faculty of Health Sciences, University of Cape Town, Cape Town, South Africa., Batista Silva Júnior J; Brazilian Health Regulatory Agency-ANVISA, Brasília, DF, Brazil.; Department of Pharmaceutical Sciences, Faculty of Health Sciences, University of Brasília, Brasília, DF, Brazil., McCune JM; HIV Frontiers, Global Health Accelerator, Bill & Melinda Gates Foundation, Seattle, WA, USA.

Publikováno v: Science translational medicine [Sci Transl Med] 2024 May 08; Vol. 16 (746), pp. eadn1902. Date of Electronic Publication: 2024 May 08.

Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI).

Autor: Adair JE; Fred Hutchinson Cancer Research Center, Seattle, WA, USA. jadair@fredhutch.org.; University of Washington, Seattle, WA, USA. jadair@fredhutch.org., Androski L; Roivant Social Ventures, New York, NY, USA., Bayigga L; Joint Clinical Research Centre, Kampala, Uganda., Bazira D; Georgetown University Medical Center, Washington, District of Columbia, USA., Brandon E; IQVIA, Durham, NC, USA., Dee L; AIDS Action Baltimore, Baltimore, MD, USA.; amFAR Institute for HIV Cure Community Advisory Board, New York, NY, USA.; Delaney AIDS Research Enterprise Community Advisory Board, San Francisco, CA, USA.; Martin Delaney Collaboratory Community Advisory Board, Bethesda, MD, USA., Deeks S; University of California at San Francisco, San Francisco, CA, USA., Draz M; Case Western Reserve University, Cleveland, OH, USA., Dubé K; University of North Carolina, Chapel Hill, NC, USA., Dybul M; Georgetown University Medical Center, Washington, District of Columbia, USA., Gurkan U; Case Western Reserve University, Cleveland, OH, USA., Harlow E; Ministry of Health, Kampala, Uganda., Kityo C; Joint Clinical Research Centre, Kampala, Uganda., Louella M; University of Washington, Seattle, WA, USA., Malik P; Cincinnati Children's Medical Hospital, Cincinnati, OH, USA., Mathews V; Christian Medical College, Vellore, India., McKemey A; IQVIA, Durham, NC, USA., Mugerwa H; Joint Clinical Research Centre, Kampala, Uganda., Muyanja D; Joint Clinical Research Centre, Kampala, Uganda., Olayiwola O; Case Western Reserve University, Cleveland, OH, USA., Orentas RJ; University of Washington, Seattle, WA, USA.; Caring Cross, Gaithersburg, MD, USA.; Seattle Children's Research Institute, Seattle, WA, USA., Popovski A; Roivant Social Ventures, New York, NY, USA., Sheehy J; Caring Cross, Gaithersburg, MD, USA., Ssali F; Joint Clinical Research Centre, Kampala, Uganda., Nsubuga MS; Joint Clinical Research Centre, Kampala, Uganda., Tisdale JF; National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA., Verhoeyen E; Université Côte d'Azur, INSERM, C3M, Nice, France.; CIRI, INSERM, Université Lyon, CNRS, ENS de Lyon, Lyon, France., Dropulić B; Caring Cross, Gaithersburg, MD, USA. boro.dropulic@caringcross.org.

Publikováno v: Gene therapy [Gene Ther] 2023 Apr; Vol. 30 (3-4), pp. 216-221. Date of Electronic Publication: 2021 Sep 08.