Results 151 to 160 of about 5,400 (185)

Advances in antisense oligonucleotide treatment for cancer. [PDF]

Jpn J Clin Oncol
Zhu L, Muhtar P, Goyama S, Yoshimi A.
europepmc   +1 more source

Lymphoid Organ Architecture and Hematopoiesis Disruption in Spinal Muscular Atrophy: Therapeutic Rescue by SMN Restoration. [PDF]

Int J Mol Sci
Guillamón P, Lindner G, Guillen J, Gatius A, Gras S, Martínez-España L, Piedrafita L, Panosa A, Tapia O, Mora C, Esquerda JE, Tizzano EF, Tarabal O, Calderó J.   +13 more
europepmc   +1 more source

Contaminating plasmid sequences and disrupted vector genomes in the liver following adeno-associated virus gene therapy. [PDF]

Nat Med
Buddle S, Brown LK, Morfopoulou S, Torres Montaguth OE, Scoto M, Herder V, Dhawan A, Brown JR, Atkinson L, Kopec A, Davis D, Storey N, Campos L, Sebire N, Macpherson H, Lee J, Orton R, Baranello G, Asamaphan P, Ilia G, Karda R, Belfield H, ISARIC4C Investigators, Counsell J, Waddington SN, Thomson EC, Muntoni F, Breuer J.   +27 more
europepmc   +1 more source

Loss of synaptic Munc13-1 underlies neurotransmission abnormalities in spinal muscular atrophy. [PDF]

Cell Mol Life Sci
Moradi M, Deng C, Sendtner M.
europepmc   +1 more source

The Presence of Risk and Protective HLA-DQ Haplotype Combinations and PLA2R1 Risk SNP in Hungarian Patients with Membranous Nephropathy. [PDF]

Int J Mol Sci
Bajcsi D, Maróti Z, Endreffy E, Légrády P, Ábrahám G, Iványi B.   +5 more
europepmc   +1 more source

Case Report: Spinal muscular atrophy with IgA nephropathy: a coincidence or association? [PDF]

Front Pediatr
Gu Y, Wang L, Yuan X, Han Y, Han P, Cui J, Wang X, Huang Y, Zhang L, Li C.   +9 more
europepmc   +1 more source

Liquid-liquid phase separation in neural development. [PDF]

Cell Mol Life Sci
Surini S, de Curtis I.
europepmc   +1 more source

Munc13-1 restoration mitigates presynaptic pathology in spinal muscular atrophy. [PDF]

Nat Commun
Moradi M, Weingart J, Deng C, Nasouti M, Briese M, Jablonka S, Sauer M, Sendtner M.   +7 more
europepmc   +1 more source

Biomolecular Condensates in Disease: Decoding the Material State and Engineering Precision Modulators. [PDF]

Int J Mol Sci
Han B, Li B, Wang X, Wang L.
europepmc   +1 more source

Chaperone dysfunction in motor neuron disease: new insights from studies of the SMN complex. [PDF]

Genetics
Matera AG.
europepmc   +1 more source