Results 121 to 130 of about 40,676 (197)

Improving clinical trial design for Duchenne muscular dystrophy [PDF]

, 2015

core   +1 more source

PPMO-based exon skipping therapy improves respiratory function in the <i>mdx</i> mouse model of Duchenne muscular dystrophy. [PDF]

Mol Ther Nucleic Acids
Biswas DD, Rodriguez MYH, El Haddad L, Hoffmann G, Copson E, Mayman S, Jose J, Lang S, Chen O, Dela Pena A, Levin E, Abdelbarr M, Slyne A, ElMallah MK.   +13 more
europepmc   +1 more source

Branchpoints as potential targets of exon-skipping therapies for genetic disorders. [PDF]

Mol Ther Nucleic Acids, 2023
Ohara H, Hosokawa M, Awaya T, Hagiwara A, Kurosawa R, Sako Y, Ogawa M, Ogasawara M, Noguchi S, Goto Y, Takahashi R, Nishino I, Hagiwara M.   +12 more
europepmc   +1 more source

Erratum: Therapeutic exon skipping for dysferlinopathies? [PDF]

European Journal of Human Genetics, 2010
Annemieke Aartsma-Rus, Kavita HK Singh, Ivo FAC Fokkema, Ieke B Ginjaar, Gert-Jan van Ommen, Johan T den Dunnen, Silvère M van der Maarel   +6 more
openaire   +1 more source

CRISPR-directed exon skipping; friend or foe (opportunity or warning sign)? [PDF]

Mol Ther Nucleic Acids
Banas KH, Kmiec EB.
europepmc   +1 more source

Targeting a pathogenic cryptic exon that drives HLRCC to induce exon skipping. [PDF]

Mol Ther Nucleic Acids
Maligireddy SS, Mandler MD, Lunger JC, Yuen M, Kulkarni S, Perez AR, Fitzsimmons CM, Crooks DR, Chari R, Linehan WM, Batista PJ.   +10 more
europepmc   +1 more source

Coexistence of a primary ALK-positive and MET14 exon skipping mutation double-fusion in one patient with NSCLC and response to crizotinib: A case report and literature review. [PDF]

Medicine (Baltimore)
Xu K, Wang M, Zhao J, Xu X, Song M.
europepmc   +1 more source

Severe Clinical Phenotype in Alport Syndrome Due to 2 <i>COL4A4</i> Exon-Skipping Events. [PDF]

Kidney Med
Pleško J, Kojc N, Kert Š, Petrin S, Matjašič A, Meglič A, Zupan A.   +6 more
europepmc   +1 more source

Effect of chemical modification on the exon-skipping activity of heteroduplex oligonucleotides. [PDF]

Mol Ther Nucleic Acids
Shimo T, Hasegawa J, Yoshioka K, Nakatsuji Y, Aso K, Tachibana K, Nagata T, Yokota T, Obika S.   +8 more
europepmc   +1 more source

DG9 boosts PMO nuclear uptake and exon skipping to restore dystrophic muscle and cardiac function. [PDF]

Nat Commun
Shah MNA, Wilton-Clark H, Haque F, Powell B, Sutanto LE, Maradiya R, Zhabyeyev P, Roshmi RR, Anwar S, Aslesh T, Lim KRQ, Maruyama R, Bigot A, Young CS, Bittner S, Spencer MJ, Moulton HM, Oudit GY, Yokota T.   +18 more
europepmc   +1 more source