Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations

Authors: Lucas Tricoli, Sunetra Sase, Julia L. Hacker, Vi Pham, Maxwell Chappell, Laura Breda, Stephanie N. Hurwitz, Naoto Tanaka, Carlo Castruccio Castracani, Amaliris Guerra, Zhongqi Hou, Lars Schlotawa, Karthikeyan Radhakrishnan, Matthew Hogenauer, Aoife Roche, John Everett, Frederic Bushman, Peter Kurre, Rebecca Ahrens-Nicklas, Laura A. Adang, Adeline L. Vanderver, Stefano Rivella

Source: Molecular Therapy: Nucleic Acids, Vol 36, Iss 1, Pp 102464- (2025)

Subject Terms: MT: Delivery Strategies, gene therapy, gene complementation, novel lentiviral vector, metachromatic leukodystrophy, MLD, Therapeutics. Pharmacology, RM1-950

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2162253125000186; https://doaj.org/toc/2162-2531

Access URL: https://doaj.org/article/b59f946719ad4f259d2bf5d4829f0c32

Restoring hematopoietic stem and progenitor cell function in Fancc−/− mice by in situ delivery of RNA lipid nanoparticles

Authors: Omar Banda, Sarah E. Adams, Linah Omer, Seul K. Jung, Hooda Said, Theerapat Phoka, Ying Tam, Drew Weissman, Stefano Rivella, Mohamad-Gabriel Alameh, Peter Kurre

Source: Molecular Therapy: Nucleic Acids, Vol 36, Iss 1, Pp 102423- (2025)

Subject Terms: MT: Delivery Strategies, lipid nanoparticles, Fanconi anemia, monogenic disorders, protein replacement therapy, in vivo delivery, Therapeutics. Pharmacology, RM1-950

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S216225312400310X; https://doaj.org/toc/2162-2531

Access URL: https://doaj.org/article/7b56694ebda2443899bf8a30b787630b

Dynamic tracking of native precursors in adult mice

Authors: Suying Liu, Sarah E Adams, Haotian Zheng, Juliana Ehnot, Seul K Jung, Greer Jeffrey, Theresa Menna, Louise Purton, Hongzhe Lee, Peter Kurre

Source: eLife, Vol 13 (2024)

Subject Terms: hematopoiesis, clonal diversity, Fanconi Anemia, Medicine, Science, Biology (General), QH301-705.5

File Description: electronic resource

Relation: https://elifesciences.org/articles/97504; https://doaj.org/toc/2050-084X

Access URL: https://doaj.org/article/2fec09e563a141b8b74c7ab47651be0e

Deregulated protein homeostasis constrains fetal hematopoietic stem cell pool expansion in Fanconi anemia

Authors: Narasaiah Kovuru, Makiko Mochizuki-Kashio, Theresa Menna, Greer Jeffrey, Yuning Hong, Young me Yoon, Zhe Zhang, Peter Kurre

Source: Nature Communications, Vol 15, Iss 1, Pp 1-15 (2024)

Subject Terms: Science

File Description: electronic resource

Relation: https://doaj.org/toc/2041-1723

Access URL: https://doaj.org/article/1139306fd82a425d9bdafd123ec40c60

Replication stress increases mitochondrial metabolism and mitophagy in FANCD2 deficient fetal liver hematopoietic stem cells

Authors: Makiko Mochizuki-Kashio, Noriko Otsuki, Kota Fujiki, Sherif Abdelhamd, Peter Kurre, Markus Grompe, Atsushi Iwama, Kayoko Saito, Ayako Nakamura-Ishizu

Source: Frontiers in Oncology, Vol 13 (2023)

Subject Terms: Hematopoietic stem cell, FANCD2, replication stress, mitochondria metabolism, mitophagy, fetal liver, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282

File Description: electronic resource

Relation: https://www.frontiersin.org/articles/10.3389/fonc.2023.1108430/full; https://doaj.org/toc/2234-943X

Access URL: https://doaj.org/article/1549e69d8d5b4516bf6cdb405a36854c

Current challenges and future directions for engineering extracellular vesicles for heart, lung, blood and sleep diseases

Authors: Guoping Li, Tianji Chen, James Dahlman, Lola Eniola‐Adefeso, Ionita C. Ghiran, Peter Kurre, Wilbur A. Lam, Jennifer K. Lang, Eduardo Marbán, Pilar Martín, Stefan Momma, Malcolm Moos, Deborah J. Nelson, Robert L. Raffai, Xi Ren, Joost P. G. Sluijter, Shannon L. Stott, Gordana Vunjak‐Novakovic, Nykia D. Walker, Zhenjia Wang, Kenneth W. Witwer, Phillip C. Yang, Martha S. Lundberg, Margaret J. Ochocinska, Renee Wong, Guofei Zhou, Stephen Y. Chan, Saumya Das, Prithu Sundd

Source: Journal of Extracellular Vesicles, Vol 12, Iss 2, Pp n/a-n/a (2023)

Subject Terms: Heart, lung, blood and sleep (HLBS) diseases, extracellular vesicles (EVs), therapeutics and diagnostics, Cytology, QH573-671

File Description: electronic resource

Relation: https://doaj.org/toc/2001-3078

Access URL: https://doaj.org/article/18fab64d303f4b96a14f506db0bcec94

Extracellular Vesicles and Chemotherapy Resistance in the AML Microenvironment

Authors: Jill Nehrbas, John T. Butler, Ding-Wen Chen, Peter Kurre

Source: Frontiers in Oncology, Vol 10 (2020)

Subject Terms: acute myeloid leukemia, extracellular vesicles, chemoresistance, bone marrow microenvironment, stroma, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282

File Description: electronic resource

Relation: https://www.frontiersin.org/article/10.3389/fonc.2020.00090/full; https://doaj.org/toc/2234-943X

Access URL: https://doaj.org/article/20e2977338db4c71a9fe6c33dd4eed83

Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study

Authors: Zora R. Rogers, Taizo A. Nakano, Timothy S. Olson, Alison A. Bertuch, Winfred Wang, Alfred Gillio, Thomas D. Coates, Anjulika Chawla, Paul Castillo, Peter Kurre, Christopher Gamper, Carolyn M. Bennett, Sarita Joshi, Amy E. Geddis, Jessica Boklan, Grzegorz Nalepa, Jennifer A. Rothman, James N. Huang, Gary M. Kupfer, Michaela Cada, Bertil Glader, Kelly J. Walkovich, Alexis A. Thompson, Rabi Hanna, Adrianna Vlachos, Maggie Malsch, Edie A. Weller, David A. Williams, Akiko Shimamura

Source: Haematologica, Vol 104, Iss 10 (2019)

Subject Terms: Diseases of the blood and blood-forming organs, RC633-647.5

File Description: electronic resource

Relation: https://haematologica.org/article/view/9081; https://doaj.org/toc/0390-6078; https://doaj.org/toc/1592-8721

Access URL: https://doaj.org/article/e30ee0ec051d4c8a8105c5d827063f09

Endogenous DNA Damage Leads to p53-Independent Deficits in Replicative Fitness in Fetal Murine Fancd2−/− Hematopoietic Stem and Progenitor Cells

Authors: Young me Yoon, Kelsie J. Storm, Ashley N. Kamimae-Lanning, Natalya A. Goloviznina, Peter Kurre

Source: Stem Cell Reports, Vol 7, Iss 5, Pp 840-853 (2016)

Subject Terms: Fanconi anemia, hematopoiesis, stem cells, development, bone marrow failure, Medicine (General), R5-920, Biology (General), QH301-705.5

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2213671116302119; https://doaj.org/toc/2213-6711

Access URL: https://doaj.org/article/4542a0378d944d69814b1ee2a0b7eba7

Phenotypic correction of Fanconi anemia cells in the murine bone marrow after carrier cell mediated delivery of lentiviral vector

Authors: Santhosh Chakkaramakkil Verghese, Natalya A. Goloviznina, Peter Kurre

Source: Stem Cell Research & Therapy, Vol 7, Iss 1, Pp 1-7 (2016)

Subject Terms: Fanconi Anemia, Gene Therapy, In situ gene delivery, Lentiviral vector, Hematopoietic stem cells, FA gene therapy, Medicine (General), R5-920, Biochemistry, QD415-436

File Description: electronic resource

Relation: http://link.springer.com/article/10.1186/s13287-016-0431-z; https://doaj.org/toc/1757-6512

Access URL: https://doaj.org/article/4e6af83420734ddfa052bf1e04a89601

Extracellular vesicles in the hematopoietic microenvironment

Authors: John T. Butler, Sherif Abdelhamed, Peter Kurre

Source: Haematologica, Vol 103, Iss 3 (2018)

Subject Terms: Diseases of the blood and blood-forming organs, RC633-647.5

File Description: electronic resource

Relation: https://haematologica.org/article/view/8376; https://doaj.org/toc/0390-6078; https://doaj.org/toc/1592-8721

Access URL: https://doaj.org/article/b99ba54262f04008b15c5fbfaad093f4

Cell-cell transmission of VSV-G pseudotyped lentivector particles.

Authors: Amy M Skinner, Santhosh Chakkaramakkil Verghese, Peter Kurre

Source: PLoS ONE, Vol 8, Iss 9, p e74925 (2013)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: http://europepmc.org/articles/PMC3769293?pdf=render; https://doaj.org/toc/1932-6203

Access URL: https://doaj.org/article/1caf544a84ec484e8b61f35746e8d159

Costs and consequences of immunosuppressive therapy in children with aplastic anemia

Authors: Evan B. Shereck, Rebecca J. Deyell, Peter Kurre

Source: Haematologica, Vol 96, Iss 6 (2011)

Subject Terms: Diseases of the blood and blood-forming organs, RC633-647.5

File Description: electronic resource

Relation: https://haematologica.org/article/view/5982; https://doaj.org/toc/0390-6078; https://doaj.org/toc/1592-8721

Access URL: https://doaj.org/article/3d87cc5cb056495abad95c1efbee679f

Cellular microvesicle pathways can be targeted to transfer genetic information between non-immune cells.

Authors: Amy M Skinner, S Lee O'Neill, Peter Kurre

Source: PLoS ONE, Vol 4, Iss 7, p e6219 (2009)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: http://europepmc.org/articles/PMC2704871?pdf=render; https://doaj.org/toc/1932-6203

Access URL: https://doaj.org/article/7e4d534834a54f689c89278f2a7995c1

Unresolved S-Phase Delays in FA HSC Lead to Deficits in Fetal Liver Hematopoietic Stem Cell Expansion in Fanconi KO Cells

Authors: Mochizuki-Kashio, Makiko, Yoon, Young me, Peter, Kurre

Source: Experimental hematology. 64(S):S87-S87

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100081610131.0x000001&indx=1&recIds=ETOCvdc_100081610131.0x000001

3117 - Unresolved S-Phase Delays in FA HSC Lead to Deficits in Fetal Liver Hematopoietic Stem Cell Expansion in Fanconi KO Cells

Authors: Mochizuki-Kashio, Makiko, Yoon, Young me, Peter, Kurre

Source: In Experimental Hematology August 2018 64 Supplement:S87-S87

Multiarm High-Throughput Integration Site Detection Limitations of LAM-PCR Technology and Optimization for Clonal Analysis.

Authors: Michael A. Harkey, Rajinder Kaul, Michael A. Jacobs, Peter Kurre, Don Bovee, Ruth Levy, C. Anthony Blau

Source: Stem Cells & Development; Jun2007, Vol. 16 Issue 3, p381-392, 12p

Reduced Toxicity and Prompt Engraftment After Minimal Conditioning of a Patient With Fanconi Anemia Undergoing Hematopoietic Stem Cell Transplantation From an HLA-Matched Unrelated Donor.

Authors: Peter Kurre, Michael Pulsipher, Ann Woolfrey, Michael Maris, Brenda Sandmaier, Hans-Peter Kiem, Rainier Storb

Source: Journal of Pediatric Hematology/Oncology; Jul2003, Vol. 25 Issue 7, p581, 3p

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