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@ARTICLE{Shibuya:163738,
      author       = {Shibuya, Yohei and Kumar, Kevin K and Mader, Marius
                      Marc-Daniel and Yoo, Yongjin and Ayala, Luis Angel and Zhou,
                      Mu and Mohr, Manuel Alexander and Neumayer, Gernot and
                      Kumar, Ishan and Yamamoto, Ryo and Marcoux, Paul and Liou,
                      Benjamin and Bennett, F Chris and Nakauchi, Hiromitsu and
                      Sun, Ying and Chen, Xiaoke and Heppner, Frank and
                      Wyss-Coray, Tony and Südhof, Thomas C and Wernig, Marius},
      title        = {{T}reatment of a genetic brain disease by {CNS}-wide
                      microglia replacement.},
      journal      = {Science translational medicine},
      volume       = {14},
      number       = {636},
      issn         = {1946-6234},
      address      = {Washington, DC},
      publisher    = {AAAS},
      reportid     = {DZNE-2022-00477},
      pages        = {eabl9945},
      year         = {2022},
      abstract     = {Hematopoietic cell transplantation after myeloablative
                      conditioning has been used to treat various genetic
                      metabolic syndromes but is largely ineffective in diseases
                      affecting the brain presumably due to poor and variable
                      myeloid cell incorporation into the central nervous system.
                      Here, we developed and characterized a near-complete and
                      homogeneous replacement of microglia with bone marrow cells
                      in mice without the need for genetic manipulation of donor
                      or host. The high chimerism resulted from a competitive
                      advantage of scarce donor cells during microglia
                      repopulation rather than enhanced recruitment from the
                      periphery. Hematopoietic stem cells, but not immediate
                      myeloid or monocyte progenitor cells, contained full
                      microglia replacement potency equivalent to whole bone
                      marrow. To explore its therapeutic potential, we applied
                      microglia replacement to a mouse model for Prosaposin
                      deficiency, which is characterized by a progressive
                      neurodegeneration phenotype. We found a reduction of
                      cerebellar neurodegeneration and gliosis in treated brains,
                      improvement of motor and balance impairment, and life span
                      extension even with treatment started in young adulthood.
                      This proof-of-concept study suggests that efficient
                      microglia replacement may have therapeutic efficacy for a
                      variety of neurological diseases.},
      keywords     = {Animals / Bone Marrow Cells / Brain / Brain Diseases /
                      Central Nervous System / Hematopoietic Stem Cell
                      Transplantation / Mice / Microglia},
      cin          = {AG Heppner},
      ddc          = {500},
      cid          = {I:(DE-2719)1810007},
      pnm          = {353 - Clinical and Health Care Research (POF4-353)},
      pid          = {G:(DE-HGF)POF4-353},
      typ          = {PUB:(DE-HGF)16},
      pmc          = {pmc:PMC9618306},
      pubmed       = {pmid:35294256},
      doi          = {10.1126/scitranslmed.abl9945},
      url          = {https://pub.dzne.de/record/163738},
}