Results 121 to 130 of about 5,752 (150)

Mucopolysaccharidosis type IVA and severe hidradenitis suppurativa: A case series. [PDF]

JAAD Case Rep
Fialová J, Kojanová M, Šáhó R, Magner M.   +3 more
europepmc   +1 more source

Lentiviral Vector-Mediated <i>Ex Vivo</i> Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis IVA Murine Model. [PDF]

Hum Gene Ther
Celik B, Rintz E, Sansanwal N, Khan S, Bigger B, Tomatsu S.   +5 more
europepmc   +1 more source

Mesenchymal Stem Cell-Derived Extracellular Vesicles: Seeking into Cell-Free Therapies for Bone-Affected Lysosomal Storage Disorders. [PDF]

Int J Mol Sci
Leal AF, Pachajoa H, Tomatsu S.
europepmc   +1 more source

CRISPR/Cas-Based Ex Vivo Gene Therapy and Lysosomal Storage Disorders: A Perspective Beyond Cas9. [PDF]

Cells
Leal AF, Prieto LE, Pachajoa H.
europepmc   +1 more source

Allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis patients: a single-center experience and assessment of quality of life. [PDF]

Ital J Pediatr
Zhang W, Huang Y, Su X, Zhao X, Sheng H, Liang C, Jiang M, Zeng C, Cai Y, Lin Y, Shao Y, Liu S, Jiang H, Liu L.   +13 more
europepmc   +1 more source

Epigenetic landscape in lysosomal storage disorders: mechanisms and modulation. [PDF]

Front Genet
Leal AF, Pachajoa H, Tomatsu S.
europepmc   +1 more source

Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice. [PDF]

Hum Gene Ther
Herreño-Pachón AM, Sawamoto K, Stapleton M, Khan S, Piechnik M, Álvarez JV, Tomatsu S.   +6 more
europepmc   +1 more source

Case report: Successful treatment of a patient presenting with a very rare association of acute lymphoblastic leukemia and mucopolysaccharidosis type IVA. [PDF]

Front Pediatr
Arnaboldi SMC, Faraguna MC, Colombini A, Sala A, Leoni V, Spinelli M, Gotti G, Bettini LR, Crescitelli V, Commone A, Gasperini S, Rizzari C.   +11 more
europepmc   +1 more source

Advances in mucopolysaccharidosis research: the impact of mass spectrometry-based approaches. [PDF]

Clin Proteomics
Ramarajan MG, Garapati K, Ghose V, Pandey A.   +3 more
europepmc   +1 more source

Alzheimer's Tau seeds-induced pathology enhances hippocampal extracellular diffusion. [PDF]

Commun Biol
Estaún-Panzano J, Lovisotto A, Mazzocco C, Piva C, Darricau M, Calaresu I, Gresil Q, Planche V, Dehay B, Groc L, Cognet L, Bezard E.   +11 more
europepmc   +1 more source