Suchergebnisse - "Bennett, C"

PubPharm (147)

1

Antisense drugs for rare and ultra-rare genetic neurological diseases

enthalten in: Neuron | 2023

von McCauley, M. | Bennett, C.

2

Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer's disease : a phase 1b, randomized, placebo-controlled trial

enthalten in: Nature medicine | 2023

von Mummery, C. | Börjesson-Hanson, A. | Blackburn, D. | +24

ErratumIn: Nat Med. 2024 Jan;30(1):304. - PMID 37845513

3

Personalized antisense oligonucleotides 'for free, for life' - the n-Lorem Foundation

enthalten in: Nature medicine | 2023

von Gleeson, J. | Bennett, C. | Carroll, J. | +6

4

Mechanism of STMN2 cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies

enthalten in: Science (New York, N.Y.) | 2023

von Baughn, M. | Melamed, Z. | López-Erauskin, J. | +21

CommentIn: Nat Rev Neurol. 2023 Jun;19(6):326. - PMID 37012364

5

Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1 : a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial

enthalten in: The Lancet. Neurology | 2023

von Thornton, C. | Moxley, R. | Eichinger, K. | +24

CommentIn: Lancet Neurol. 2023 Mar;22(3):191-192. - PMID 36804078

6

Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1

enthalten in: Gene therapy | 2022

von Ait Benichou, S. | Jauvin, D. | De Serres-Bérard, T. | +7

7

Preclinical and Phase 1 Assessment of Antisense Oligonucleotide Bepirovirsen in Hepatitis B Virus-Transgenic Mice and Healthy Human Volunteers : Support for Clinical Dose Selection and Evaluation of Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses

enthalten in: Clinical pharmacology in drug development | 2022

von Han, K. | Theodore, D. | McMullen, G. | +8

8

Combinatorial ASO-mediated therapy with low dose SMN and the protective modifier Chp1 is not sufficient to ameliorate SMA pathology hallmarks

enthalten in: Neurobiology of disease | 2022

von Muinos-Bühl, A. | Rombo, R. | Janzen, E. | +5

9

ASO-Based PKM Splice-Switching Therapy Inhibits Hepatocellular Carcinoma Growth

enthalten in: Cancer research | 2022

von Ma, W. | Voss, D. | Scharner, J. | +8

10

Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood-brain barrier and knock down genes in the rodent CNS

enthalten in: Nature biotechnology | 2021

von Nagata, T. | Dwyer, C. | Yoshida-Tanaka, K. | +21

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