Browse by UCL people
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Number of items: 28.
Article
Aguti, S;
Bolduc, V;
Ala, P;
Turmaine, M;
Bönnemann, CG;
Muntoni, F;
Zhou, H;
(2020)
Exon-Skipping Oligonucleotides Restore Functional Collagen VI by Correcting a Common COL6A1 Mutation in Ullrich CMD.
Molecular Therapy - Nucleic Acids
, 21
pp. 205-216.
10.1016/j.omtn.2020.05.029.
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Aguti, S;
Malerba, A;
Zhou, H;
(2018)
The progress of AAV-mediated gene therapy in neuromuscular disorders.
Expert Opinion on Biological Therapy
, 18
(6)
pp. 681-693.
10.1080/14712598.2018.1479739.
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Bernabò, P;
Tebaldi, T;
Groen, EJN;
Lane, FM;
Perenthaler, E;
Mattedi, F;
Newbery, HJ;
... Viero, G; + view all
(2017)
In Vivo Translatome Profiling in Spinal Muscular Atrophy Reveals a Role for SMN Protein in Ribosome Biology.
Cell Reports
, 21
(4)
pp. 953-965.
10.1016/j.celrep.2017.10.010.
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Bolduc, V;
Foley, AR;
Solomon-Degefa, H;
Sarathy, A;
Donkervoort, S;
Hu, Y;
Chen, GS;
... Bönnemann, CG; + view all
(2019)
A recurrent COL6A1 pseudoexon insertion causes muscular dystrophy and is effectively targeted by splice-correction therapies.
JCI Insight
, 4
(6)
10.1172/jci.insight.124403.
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Catapano, F;
Zaharieva, I;
Scoto, M;
Marrosu, E;
Morgan, J;
Muntoni, F;
Zhou, H;
(2016)
Altered Levels of MicroRNA-9,-206, and-132 in Spinal Muscular Atrophy and Their Response to Antisense Oligonucleotide Therapy.
Molecular Therapy-Nucleic Acids
, 5
, Article e331. 10.1038/mtna.2016.47.
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Hammond, SM;
Hazell, G;
Shabanpoor, F;
Saleh, AF;
Bowerman, M;
Sleigh, JN;
Meijboom, KE;
... Wood, MJ; + view all
(2016)
Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.
Proceedings of the National Academy of Sciences of the United States of America
, 113
(39)
pp. 10962-10967.
10.1073/pnas.1605731113.
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Lopez, RJ;
Byrne, S;
Vukcevic, M;
Sekulic-Jablanovic, M;
Xu, L;
Brink, M;
Alamelu, J;
... Jungbluth, H; + view all
(2016)
An RYR1 mutation associated with malignant hyperthermia is also associated with bleeding abnormalities.
Science Signaling
, 9
(435)
, Article ra68. 10.1126/scisignal.aad9813.
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Marrosu, E;
Ala, P;
Muntoni, F;
Zhou, H;
(2017)
Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy.
Molecular Therapy - Nucleic Acids
, 8
pp. 416-427.
10.1016/j.omtn.2017.07.006.
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Mitrpant, C;
Porensky, P;
Zhou, H;
Price, L;
Muntoni, F;
Fletcher, S;
Wilton, SD;
(2013)
Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy.
PLoS One
, 8
(4)
, Article e62114. 10.1371/journal.pone.0062114.
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Owen, N;
Zhou, HY;
Malygin, AA;
Sangha, J;
Smith, LD;
Muntoni, F;
Eperon, IC;
(2011)
Design principles for bifunctional targeted oligonucleotide enhancers of splicing.
Nucleic Acids Research
, 39
(16)
7194- 7208.
10.1093/nar/gkr152.
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Rahim, Ahad A;
Kurian, Manju A;
Zhou, Haiyan;
Ferguson, Ross;
Tabrizi, Sarah J;
Lignani, Gabriele;
Aquilina, Kristian;
(2025)
Genetic therapies for neurological diseases.
Pharmacological Reviews
, Article 100093. 10.1016/j.pharmr.2025.100093.
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Sardone, V;
Zhou, H;
Muntoni, F;
Ferlini, A;
Falzarano, MS;
(2017)
Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease.
Molecules
, 22
(4)
, Article 563. 10.3390/molecules22040563.
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Sintusek, P;
Catapano, F;
Angkathunkayul, N;
Marrosu, E;
Parson, SH;
Morgan, JE;
Muntoni, F;
(2016)
Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment.
PLOS One
, 11
(5)
, Article e0155032. 10.1371/journal.pone.0155032.
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Song, Huiling;
Li, Haosheng;
Shen, Xiaonan;
Liu, Kuai;
Feng, Haoran;
Cui, Jiahua;
Wei, Wei;
... Du, Zixiu; + view all
(2024)
A pH-responsive cetuximab-conjugated DMAKO-20 nano-delivery system for overcoming K-ras mutations and drug resistance in colorectal carcinoma.
Acta Biomaterialia
, 177
pp. 456-471.
10.1016/j.actbio.2024.01.047.
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Spicer, C;
Lu, C-H;
Catapano, F;
Scoto, M;
Zaharieva, I;
Malaspina, A;
Morgan, JE;
... Zhou, H; + view all
(2021)
The altered expression of neurofilament in mouse models and patients with spinal muscular atrophy.
Annals of Clinical and Translational Neurology
10.1002/acn3.51336.
(In press).
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Szunyogova, E;
Zhou, H;
Maxwell, GK;
Powis, RA;
Francesco, M;
Gillingwater, TH;
Parson, SH;
(2016)
Survival Motor Neuron (SMN) protein is required for normal mouse liver development.
SCIENTIFIC REPORTS
, 6
(ARTN 346)
10.1038/srep34635.
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Tagalakis, AD;
Castellaro, S;
Zhou, H;
Bienemann, A;
Munye, MM;
McCarthy, D;
White, EA;
(2015)
A method for concentrating lipid peptide DNA and siRNA nanocomplexes that retains their structure and transfection efficiency.
Int J Nanomedicine
, 10
2673 - 2683.
10.2147/IJN.S78935.
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Tagalakis, AD;
Lee, DH;
Bienemann, AS;
Zhou, H;
Munye, MM;
Saraiva, L;
McCarthy, D;
... Hart, SL; + view all
(2014)
Multifunctional, self-assembling anionic peptide-lipid nanocomplexes for targeted siRNA delivery.
Biomaterials
, 35
(29)
pp. 8406-8415.
10.1016/j.biomaterials.2014.06.003.
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Torres-Masjoan, Laia;
Aguti, Sara;
Zhou, Haiyan;
Muntoni, Francesco;
(2025)
Clinical applications of exon skipping antisense oligonucleotides in neuromuscular diseases.
Molecular Therapy
10.1016/j.ymthe.2025.04.038.
(In press).
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Zaharieva, Irina T;
Scoto, Mariacristina;
Aragon‐Gawinska, Karolina;
Ridout, Deborah;
Doreste, Bruno;
Servais, Laurent;
Muntoni, Francesco;
(2022)
Response of plasma microRNAs
to nusinersen treatment in patients with
SMA.
Annals of Clinical and Translational Neurology
10.1002/acn3.51579.
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Zhou, Haiyan;
Arechavala-Gomeza, Virginia;
Garanto, Alejandro;
(2023)
Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics.
Nucleic Acid Therapeutics
10.1089/nat.2023.0001.
(In press).
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Zhou, Haiyan;
Hong, Ying;
Scoto, Mariacristina;
Thomson, Alison;
Pead, Emma;
MacGillivray, Tom;
Hernandez-Gerez, Elena;
... Muntoni, Francesco; + view all
(2022)
Microvasculopathy in SMA is driven by a reversible autonomous endothelial cell defect.
Journal of Clinical Investigation
, 132
(21)
, Article e153430. 10.1172/JCI153430.
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Zhou, H;
(2020)
Application and Mechanism of Action of Antisense Oligonucleotides in Precise Medicine.
Journal of Precision Medicine
, 35
(4)
pp. 283-286.
10.13362/j.jpmed.202004001.
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Zhou, H;
Meng, J;
Malerba, A;
Catapano, F;
Sintusek, P;
Jarmin, S;
Feng, L;
... Muntoni, F; + view all
(2020)
Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy.
Journal of Cachexia, Sarcopenia and Muscle
10.1002/jcsm.12542.
(In press).
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Zhou, H;
Muntoni, F;
(2020)
The authors reply: Letter on: "Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy" by Zhou et al .
[Letter].
Journal of Cachexia, Sarcopenia and Muscle
10.1002/jcsm.12608.
(In press).
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Zhou, H;
Muntoni, F;
(2018)
Morpholino-Mediated Exon Inclusion for SMA.
Exon Skipping and Inclusion Therapies
, 1828
pp. 467-477.
10.1007/978-1-4939-8651-4_29.
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Zhou, H;
Rokach, O;
Feng, L;
Munteanu, I;
Mamchaoui, K;
Wilmshurst, JM;
Sewry, C;
... Muntoni, F; + view all
(2013)
RyR1 Deficiency in Congenital Myopathies Disrupts Excitation-Contraction Coupling.
Human Mutation
, 34
(7)
pp. 986-996.
10.1002/humu.22326.
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Book chapter
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Aguti, Sara;
Guirguis, Fady;
Bönnemann, Carsten;
Muntoni, Francesco;
Bolduc, Veronique;
Zhou, Haiyan;
(2022)
Exon-Skipping for a Pathogenic COL6A1 Variant in Ullrich Congenital Muscular Dystrophy.
In: Maruyama, R and Yokota, T, (eds.)
Methods in Molecular Biology.
(pp. 387-407).
Springer Nature: New York, NY, USA.
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