Zobrazeno 1 - 10
of 23
pro vyhledávání: '"Dakai Mu"'
Autor:
Bence György, Camilla Lööv, Mikołaj P. Zaborowski, Shuko Takeda, Benjamin P. Kleinstiver, Caitlin Commins, Ksenia Kastanenka, Dakai Mu, Adrienn Volak, Vilmantas Giedraitis, Lars Lannfelt, Casey A. Maguire, J. Keith Joung, Bradley T. Hyman, Xandra O. Breakefield, Martin Ingelsson
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 11, Iss C, Pp 429-440 (2018)
The APPswe (Swedish) mutation in the amyloid precursor protein (APP) gene causes dominantly inherited Alzheimer’s disease (AD) as a result of increased β-secretase cleavage of the amyloid-β (Aβ) precursor protein. This leads to abnormally high A
Externí odkaz:
https://doaj.org/article/02b58cb34d3c4ec3bd5ead3c96a5fabe
Autor:
Jonathan Dashkoff, Eli P Lerner, Nhi Truong, Jacob A Klickstein, Zhanyun Fan, Dakai Mu, Casey A Maguire, Bradley T Hyman, Eloise Hudry
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
The capacity of certain adeno-associated virus (AAV) vectors to cross the blood–brain barrier after intravenous delivery offers a unique opportunity for noninvasive brain delivery. However, without a well-tailored system, the use of a peripheral ro
Externí odkaz:
https://doaj.org/article/268aaa1355464350a8486123556bc6b4
Autor:
Alain Brisson, Sisareuth Tan, David P. Corey, Adrienn Volak, Zachary Fitzpatrick, Xandra O. Breakefield, Maria Ericsson, Casey A. Maguire, Panos I. Tamvakologos, Deborah I. Scheffer, Dakai Mu, Artur A. Indzhykulian, Xudong Wu, Bence György, Cyrille Sage, Yaqiao Li
Publikováno v:
Molecular Therapy. 25:379-391
Adeno-associated virus (AAV) is a safe and effective vector for gene therapy for retinal disorders. Gene therapy for hearing disorders is not as advanced, in part because gene delivery to sensory hair cells of the inner ear is inefficient. Although A
Autor:
Servio H. Ramirez, Bradley T. Hyman, Dakai Mu, Evan M. Lutton, Allison M. Andrews, Steven F. Merkel, Eloise Hudry, Casey A. Maguire
Publikováno v:
Journal of Neurochemistry. 140:216-230
Developing therapies for central nervous system (CNS) diseases is exceedingly difficult because of the blood–brain barrier (BBB). Notably, emerging technologies may provide promising new options for the treatment of CNS disorders. Adeno-associated
Autor:
Dwijit GuhaSarkar, Dakai Mu, Bradley T. Hyman, Casey A. Maguire, Kelsey Pinkham, Miguel Sena-Esteves, Jeya Shree Natasan, Zachary Fitzpatrick, Pike See Cheah, Adrienn Volak, Stanley G. LeRoy, Sheetal Gandhi, Christian E. Badr, Andreas Maus, David J. Park, Anat Stemmer-Rachamimov, Eloise Hudry
The malignant primary brain tumor, glioblastoma (GBM) is generally incurable. New approaches are desperately needed. Adeno-associated virus (AAV) vector-mediated delivery of anti-tumor transgenes is a promising strategy, however direct injection lead
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b2ae4be9d884a2d1b94e538b045f8095
https://europepmc.org/articles/PMC6454875/
https://europepmc.org/articles/PMC6454875/
Autor:
Ann B. Moser, Florian Eichler, Xandra O. Breakefield, Patricia L. Musolino, Dakai Mu, Casey A. Maguire, Jia Qian Ren, Yi Gong, Shilpa Prabhakar
Publikováno v:
Molecular Therapy. 23:824-834
X-linked adrenoleukodystrophy (X-ALD) is a devastating neurological disorder caused by mutations in the ABCD1 gene that encodes a peroxisomal ATP-binding cassette transporter (ABCD1) responsible for transport of CoA-activated very long-chain fatty ac
Autor:
Henia Dar, Daniel Henderson, Zinkal Padalia, Ashley Porras, Dakai Mu, Maeng Kyungah, Seshidhar Police, Demetrios Kalaitzidis, Jonathan Terrett, Jason Sagert
Publikováno v:
Blood. 132:1921-1921
Autologous CAR-T cells targeting BCMA have induced robust and durable responses in patients with relapsed/refractory multiple myeloma. However, autologous cell therapies face several challenges which will likely limit the number of patients that will
Autor:
Steven F. Merkel, Servio H. Ramirez, Hemi Dimant, Bradley T. Hyman, Eloise Hudry, Sisareuth Tan, Bence György, Sheetal Gandhi, Deborah I. Scheffer, Casey A. Maguire, Federico Mingozzi, Zachary Fitzpatrick, Alain Brisson, Dakai Mu, Marissa Masek, Tim Ragan, Courtney Martin
Publikováno v:
Gene Ther
Gene Ther, 2016, 23 (4), pp.380-92. ⟨10.1038/gt.2016.11⟩
Gene therapy
Gene Ther, 2016, 23 (4), pp.380-92. ⟨10.1038/gt.2016.11⟩
Gene therapy
International audience; Adeno-associated virus (AAV) vectors are showing promise in gene therapy trials and have proven to be extremely efficient biological tools in basic neuroscience research. One major limitation to their widespread use in the neu
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d6c260596a625badcf11a629de3885b0
https://hal.archives-ouvertes.fr/hal-02880989
https://hal.archives-ouvertes.fr/hal-02880989
Autor:
Zinkal Padalia, Ashley Porras, Mary Lee Dequeant, Jason Sagert, Thao Nguyen, Matthias John, Melanie Allen, Henia Dar, Daniel Henderson, Seshidar Police, Dakai Mu, Kelly Maeng, Elaine Huang, Sarah Spencer, Nickolaus Lorson, Paul Gonzales, Chelsea Holmquist, Gregg Hirschfeld, Jonathan A. Terrett, Demetrios Kalaitzidis
Publikováno v:
Cancer Research. 78:2551-2551
Autologous CAR T therapeutics have recently been approved for use in B-cell malignancies. While responses have been impressive using CD19 directed CAR T cells there has been a lack of comparable success for CAR T cells directed at solid tumor antigen
Autor:
Henia Dar, Daniel Henderson, Zinkal Padalia, Ashley Porras, Dakai Mu, Kyungah Maeng, Seshi Police, Demetrios Kalaitzidis, Jonathan Terrett, Jason Sagert
Publikováno v:
Cancer Research. 78:1540-1540
B cell maturation antigen (BCMA) is a tumor necrosis factor family cell surface receptor that binds B cell activating factor (BAFF) and a proliferation inducing ligand (APRIL) and is involved in the long-term survival of B cells. BCMA has been shown