Results 81 to 90 of about 6,342 (125)

Diagnostic and therapeutic applications of the glycan biomarker H3N2b in GM1 Gangliosidosis. [PDF]

Mol Genet Metab
Kell P, Mishra S, D'Souza P, Tifft CJ, Dietzen DJ, Lasio LD, Carson JP, Hong X, Schiffmann R, Ledesma MD, Tayebi N, Dickson PI, Porter FD, Ory DS, Jiang X.   +14 more
europepmc   +1 more source

Neuritin1 Cis-Regulatory Elements Enable Gene Expression Preferentially in Retinal Ganglion Cells. [PDF]

Invest Ophthalmol Vis Sci
Talla V, Koilkonda R, Kinane C, Panchal M, Khuu T, Park KK.   +5 more
europepmc   +1 more source

Mass Determination of Filled and Empty AAV5 Particles Enabled by Nanoelectrospray Ionization and Proton Transfer Charge Reduction. [PDF]

Anal Chem
Pizzala NJ, Faye BKS, Song H, Tao L, McLuckey SA.   +4 more
europepmc   +1 more source

Methodological validation of a cryo-TEM-based detection method for empty capsid ratio in recombinant adeno-associated virus. [PDF]

Biochem Biophys Rep
Xu L, Chen J, Zhu W, Zhao Y, Zheng F, Du R, Jiang Y, Yang Y, Chen Y, Dong Y, Zhang Z, Tong Y.   +11 more
europepmc   +1 more source

Recombinant adeno-associated virus 2-mediated miRNA-199 suppression vector alleviates dextran sulfate sodium-induced ulcerative colitis in mice. [PDF]

Virus Res
Shanshan W, Yu Z.
europepmc   +1 more source

Gene-replacement therapy in neurodevelopmental disorders: progress and challenges. [PDF]

J Clin Invest
Lerche H, Hedrich UB, Wuttke TV.
europepmc   +1 more source

T2 Hyperintensities in Gracile Tracts of Cervical Spinal Cord in Giant Axonal Neuropathy (GAN). [PDF]

Muscle Nerve
Armao D, Bouldin TW, Bharucha-Goebel DX, Hartman TS, Gray SJ, Bailey RM, Saade D, Todd JJ, Jain M, Waite M, Bönnemann CG, Smith JK.   +11 more
europepmc   +1 more source

Advances in endothelial cell targeting by AAV vectors. [PDF]

Acta Biochim Pol
Cichon M, Jozkowicz A, Grochot-Przeczek A.   +2 more
europepmc   +1 more source

Triple rAAV9 Vector Combinations Encoding Broadly Neutralizing Antibodies Effectively Suppress HIV-1 Infection in Humanized Mice. [PDF]

Int J Mol Sci
Leontyev DS, Urusov FA, Glazkova DV, Belugin BV, Mitiushina AA, Tsyganova GM, Yudin SM, Bogoslovskaya EV, Shipulin GA.   +8 more
europepmc   +1 more source

Frameless intraputaminal delivery of gene therapy with eladocagene exuparvovec in patients with aromatic L-amino acid decarboxylase deficiency: safe and efficient results. [PDF]

Childs Nerv Syst
Mai R, Reshchikov D, Popov V, Gorelikov S, Zakharova E, Mikhaylova S.   +5 more
europepmc   +1 more source