Browse by UCL people
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Number of items: 47.
Article
Baruteau, J;
Perocheau, DP;
Hanley, J;
Lorvellec, M;
Rocha-Ferreira, E;
Karda, R;
Ng, J;
... Waddington, SN; + view all
(2018)
Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer.
Nature Communications
, 9
(1)
, Article 3505. 10.1038/s41467-018-05972-1.
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Bhangra, KS;
Busuttil, F;
Phillips, JB;
Rahim, AA;
(2016)
Using Stem Cells to Grow Artificial Tissue for Peripheral Nerve Repair.
Stem Cells International
, 2016
, Article 7502178. 10.1155/2016/7502178.
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Buckley, SM;
Delhove, JM;
Perocheau, DP;
Karda, R;
Rahim, AA;
Howe, SJ;
Ward, NJ;
... McKay, TR; + view all
(2015)
In vivo bioimaging with tissue-specific transcription factor activated luciferase reporters.
Scientific Reports
, 5
, Article 11842. 10.1038/srep11842.
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Busuttil, F;
Rahim, AA;
Phillips, JB;
(2017)
Combining gene and stem cell therapy for peripheral nerve tissue engineering.
Stem Cells and Development
, 26
(4)
pp. 231-238.
10.1089/scd.2016.0188.
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Counsell, JR;
Karda, R;
Diaz, JA;
Carey, L;
Wiktorowicz, T;
Buckley, SMK;
Ameri, S;
... Howe, SJ; + view all
(2018)
Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice.
Molecular Therapy - Nucleic Acids
, 12
pp. 626-634.
10.1016/j.omtn.2018.07.006.
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Geard, AF;
Massaro, G;
Hughes, MP;
Arbuthnot, P;
Waddington, SN;
Rahim, AA;
(2025)
Generation and Treatment of a Novel Severe Model of Visceral Gaucher Disease by Genetic Therapy.
Pharmaceutics
, 17
(5)
, Article 650. 10.3390/pharmaceutics17050650.
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Habib, Abdella M;
Li, Shengnan;
Zhang, Chenjing;
Ji, Meijun;
Osorio, Nancy;
Penalba, Virginie;
Torres, Jesus M;
... Coste, Bertrand; + view all
(2025)
MDFIC2 is a PIEZO channel modulator that can alleviate mechanical allodynia associated with neuropathic pain.
Proceedings of the National Academy of Sciences
, 122
(45)
, Article e2512426122. 10.1073/pnas.2512426122.
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Holthaus, S-MK;
Herranz-Martin, S;
Massaro, G;
Aristorena, M;
Hoke, J;
Hughes, MP;
Maswood, R;
... Ali, RR; + view all
(2019)
Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease.
Human Molecular Genetics
, 28
(23)
pp. 3867-3879.
10.1093/hmg/ddz210.
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Howe, Geoffrey;
Bal, Mehtap;
Wasmuth, Matthew;
Massaro, G;
Rahim, Ahad;
Ali, Sadfer;
Rivera, Milena;
... Nesbeth, Darren; + view all
(2024)
An autonucleolytic suspension HEK293F host cell line for high-titre serum-free AAV5 and AAV9 production with reduced levels of DNA impurity.
Molecular Therapy - Methods and Clinical Development
, Article 101317. 10.1016/j.omtm.2024.101317.
(In press).
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Hughes, Michael Paul;
Nelvagal, Hemanth Ramesh;
Coombe-Tennant, Oliver;
Smith, Dave;
Smith, Claire;
Massaro, Giulia;
Poupon-Bejuit, Laura;
... Rahim, Ahad Abdul; + view all
(2023)
A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann–Pick Type C1 Disease.
Cells
, 12
(12)
, Article 1619. 10.3390/cells12121619.
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Hughes, MP;
Smith, DA;
Morris, L;
Fletcher, C;
Colaco, A;
Huebecker, M;
Tordo, J;
... Rahim, AA; + view all
(2018)
AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease.
Human Molecular Genetics
, 27
(17)
pp. 3079-3098.
10.1093/hmg/ddy212.
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Karda, R;
Buckley, SM;
Mattar, CN;
Ng, J;
Massaro, G;
Hughes, MP;
Kurian, MA;
... Rahim, AA; + view all
(2014)
Perinatal systemic gene delivery using adeno-associated viral vectors.
Front Mol Neurosci
, 7
, Article 89. 10.3389/fnmol.2014.00089.
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Karda, R;
Rahim, AA;
Wong, AMS;
Suff, N;
Diaz, JA;
Perocheau, DP;
Tijani, M;
... Waddington, SN; + view all
(2020)
Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors.
Scientific Reports
, 10
(1)
, Article 2121. 10.1038/s41598-020-59075-3.
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Kempthorne, Liam;
Vaizoglu, Deniz;
Cammack, Alexander J;
Carcolé, Mireia;
Roberts, Martha J;
Mikheenko, Alla;
Fisher, Alessia;
... Isaacs, Adrian M; + view all
(2025)
Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo.
Nature Communications
, 16
, Article 459. 10.1038/s41467-024-55550-x.
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Lambert, JRA;
Howe, SJ;
Rahim, AA;
Burke, DG;
Heales, SJR;
(2019)
Inhibition of Mitochondrial Complex I Impairs Release of α-Galactosidase by Jurkat Cells.
International Journal of Molecular Sciences
, 20
(18)
, Article 4349. 10.3390/ijms20184349.
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Liu, Wenfei;
Geard, Amy F;
Massaro, Giulia;
Hughes, Michael P;
Aristorena, Mikel;
Coombe-Tennant, Oliver;
Xu, Liangyuan;
... Rahim, Ahad A; + view all
(2025)
Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration.
Human Gene Therapy
, Article 10430342251403448. 10.1177/10430342251403448.
(In press).
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Liu, W;
Kleine-Holthaus, S-M;
Herranz-Martin, S;
Aristorena, M;
Mole, SE;
Smith, AJ;
Ali, RR;
(2020)
Experimental gene therapies for the NCLs.
Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease
, 1866
(9)
, Article 165772. 10.1016/j.bbadis.2020.165772.
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Ma, Yingchang;
Colic, Ines;
Muwaffak, Maha;
Rahim, Ahad A;
Brocchini, Steve;
Williams, Gareth R;
(2025)
In-situ hyaluronic acid-tyramine hydrogels prolong the release of extracellular vesicles and enhance stability.
International Journal of Pharmaceutics
, Article 125650. 10.1016/j.ijpharm.2025.125650.
(In press).
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Massaro, Giulia;
Geard, Amy F;
Nelvagal, Hemanth R;
Gore, Katrina;
Clemo, Nadine K;
Waddington, Simon N;
Rahim, Ahad A;
(2024)
Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease.
Human Molecular Genetics
, Article ddae081. 10.1093/hmg/ddae081.
(In press).
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Massaro, G;
Geard, AF;
Liu, W;
Coombe-Tennant, O;
Waddington, SN;
Baruteau, J;
Gissen, P;
(2021)
Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.
Biomolecules
, 11
(4)
, Article 611. 10.3390/biom11040611.
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Massaro, G;
Hughes, MP;
Whaler, SM;
Wallom, K-L;
Priestman, DA;
Platt, FM;
Waddington, SN;
(2020)
Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes.
Human Molecular Genetics
10.1093/hmg/ddz317.
(In press).
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Massaro, G;
Mattar, CNZ;
Wong, AMS;
Sirka, E;
Buckley, SMK;
Herbert, BR;
Karlsson, S;
... Rahim, AA; + view all
(2018)
Fetal gene therapy for neurodegenerative disease of infants.
Nature Medicine
, 24
pp. 1317-1323.
10.1038/s41591-018-0106-7.
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Mattar, CN;
Wong, AM;
Hoefer, K;
Alonso-Ferrero, ME;
Buckley, SM;
Howe, SJ;
Cooper, JD;
... Rahim, AA; + view all
(2015)
Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates.
The FASEB Journal
, 29
(9)
3876- 3888.
10.1096/fj.14-269092.
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Mole, SE;
Anderson, G;
Band, HA;
Berkovic, SF;
Cooper, JD;
Kleine Holthaus, S-M;
McKay, TR;
... Smith, AJ; + view all
(2019)
Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis.
[Review].
Lancet Neurology
, 18
(1)
pp. 107-116.
10.1016/S1474-4422(18)30368-5.
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Ng, J;
Barral, S;
Barrigon, CDLF;
Lignani, G;
Erdem, FA;
Wallings, R;
Privolizzi, R;
... Kurian, MA; + view all
(2021)
Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism.
Science Translational Medicine
, 13
(594)
, Article eaaw1564. 10.1126/scitranslmed.aaw1564.
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Nivsarkar, MS;
Buckley, SM;
Parker, AL;
Perocheau, D;
McKay, TR;
Rahim, AA;
Howe, SJ;
(2015)
Evidence for Contribution of CD4+CD25+ Regulatory T Cells in Maintaining Immune Tolerance to Human Factor IX following Perinatal Adenovirus Vector Delivery.
Journal of Immunology Research
, 2015
, Article 397879. 10.1155/2015/397879.
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Osellame, LD;
Rahim, AA;
Hargreaves, IP;
Gegg, ME;
Richard-Londt, A;
Brandner, S;
Waddington, SN;
... Duchen, MR; + view all
(2013)
Mitochondria and quality control defects in a mouse model of Gaucher Disease-links to Parkinson's Disease.
Cell Metabolism
, 17
(6)
pp. 941-953.
10.1016/j.cmet.2013.04.014.
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Plotegher, N;
Perocheau, D;
Ferrazza, R;
Massaro, G;
Bhosale, G;
Zambon, F;
Rahim, AA;
... Duchen, MR; + view all
(2020)
Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload.
Cell Death & Differentiation
, 27
pp. 1588-1603.
10.1038/s41418-019-0442-2.
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Poupon-Bejuit, L;
Hughes, MP;
Liu, W;
Geard, A;
Faour-Slika, N;
Whaler, S;
Massaro, G;
(2022)
A GLP1 receptor agonist diabetes drug ameliorates neurodegeneration in a mouse model of infantile neurometabolic disease.
Scientific Reports
, 12
(1)
, Article 13825. 10.1038/s41598-022-17338-1.
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Poupon-Bejuit, Laura;
Geard, Amy;
Millicheap, Nathan;
Rocha-Ferreira, Eridan;
Hagberg, Henrik;
Thornton, Claire;
Rahim, Ahad A;
(2024)
Diabetes drugs activate neuroprotective pathways in models of neonatal hypoxic-ischemic encephalopathy.
EMBO Molecular Medicine
10.1038/s44321-024-00079-1.
(In press).
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Poupon-Bejuit, L;
Rocha-Ferreira, E;
Thornton, C;
Hagberg, H;
Rahim, AA;
(2020)
Neuroprotective Effects of Diabetes Drugs for the Treatment of Neonatal Hypoxia-Ischemia Encephalopathy.
Frontiers in Cellular Neuroscience
, 14
, Article 112. 10.3389/fncel.2020.00112.
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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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Rahim, AA;
Gissen, P;
(2020)
Gene therapy for global brain diseases: one small step for mice, one giant leap for humans.
Brain
, 143
(7)
pp. 1964-1966.
10.1093/brain/awaa189.
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Rahim, AA;
Russell, C;
Mole, SE;
(2020)
Special edition: The NCLs/Batten disease.
Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease
, 1866
(9)
, Article 165824. 10.1016/j.bbadis.2020.165824.
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Rocha-Ferreira, E;
Poupon, L;
Zelco, A;
Leverin, A-L;
Nair, S;
Jonsdotter, A;
Carlsson, Y;
... Rahim, AA; + view all
(2018)
Neuroprotective exendin-4 enhances hypothermia therapy in a model of hypoxic-ischaemic encephalopathy.
Brain
, 141
(10)
pp. 2925-2942.
10.1093/brain/awy220.
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Rocha-Ferreira, E;
Rudge, B;
Hughes, MP;
Rahim, AA;
Hristova, M;
Robertson, NJ;
(2016)
Immediate Remote Ischemic Postconditioning Reduces Brain Nitrotyrosine Formation in a Piglet Asphyxia Model.
Oxidative Medicine and Cellular Longevity
, 2016
, Article 5763743. 10.1155/2016/5763743.
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Ruiz, AJ;
Rahim, AA;
(2019)
Bringing balance to the force-regulatable gene therapy for epilepsy.
[Editorial comment].
Gene Therapy
, 26
pp. 347-349.
10.1038/s41434-019-0083-6.
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Sartorelli, J;
Ng, J;
Rahim, AA;
Waddington, SN;
Kurian, MA;
(2025)
Genetic therapies for movement disorders - current status.
Journal of Neurology
, 272
, Article 220. 10.1007/s00415-025-12940-5.
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Seker Yilmaz, B;
Baruteau, J;
Rahim, AA;
Gissen, P;
(2020)
Clinical and Molecular Features of Early Infantile Niemann Pick Type C Disease.
International Journal of Molecular Sciences
, 21
(14)
, Article 5059. 10.3390/ijms21145059.
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Snowball, A;
Chabrol, E;
Wykes, RC;
Shekh-Ahmad, T;
Cornford, JH;
Lieb, A;
Hughes, MP;
... Schorge, S; + view all
(2019)
Epilepsy Gene Therapy Using an Engineered Potassium Channel.
Journal of Neuroscience
, 39
(16)
pp. 3159-3169.
10.1523/JNEUROSCI.1143-18.2019.
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Tordo, J;
O'Leary, C;
Antunes, ASLM;
Palomar, N;
Aldrin-Kirk, P;
Basche, M;
Bennett, A;
... Henckaerts, E; + view all
(2018)
A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.
Brain
10.1093/brain/awy126.
(In press).
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Vardi, A;
Zigdon, H;
Meshcheriakova, A;
Klein, AD;
Yaacobi, C;
Eilam, R;
Kenwood, BM;
... Futerman, AH; + view all
(2016)
Delineating pathological pathways in a chemically-induced mouse model of Gaucher disease.
The Journal of Pathology
, 239
(4)
pp. 496-509.
10.1002/path.4751.
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Victor, S;
Rocha-Ferreira, E;
Rahim, A;
Hagberg, H;
Edwards, D;
(2021)
New possibilities for neuroprotection in neonatal hypoxic-ischemic encephalopathy.
European Journal of Pediatrics
10.1007/s00431-021-04320-8.
(In press).
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Waddington, Simon N;
Peranteau, William H;
Rahim, Ahad A;
Boyle, Ashley K;
Kurian, Manju A;
Gissen, Paul;
Chan, Jerry KY;
(2023)
Fetal gene therapy.
Journal of Inherited Metabolic Disease
10.1002/jimd.12659.
(In press).
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Whiteley, Zoe;
Massaro, Giulia;
Gkogkos, Georgios;
Gavriilidis, Asterios;
Waddington, Simon N;
Rahim, Ahad A;
Craig, Duncan QM;
(2023)
Microfluidic production of nanogels as alternative triple transfection reagents for the manufacture of adeno-associated virus vectors.
Nanoscale
10.1039/d2nr06401d.
(In press).
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Book chapter
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Allahham, Nour;
Colic, Ines;
Rayner, Melissa LD;
Gurnani, Pratik;
Phillips, James B;
Rahim, Ahad A;
Williams, Gareth R;
(2023)
Advanced Formulation Approaches for Emerging Therapeutic Technologies.
In: Michel, Martin C, (ed.)
Handbook of Experimental Pharmacology.
(pp. 1-23).
Springer Verlag: Berlin, Heidelberg, Germany.
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Proceedings paper
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Karda, R;
Perocheau, DP;
Suff, N;
Ng, J;
Hughes, M;
Rahim, AA;
Buckley, SMK;
... Waddington, SN; + view all
(2016)
Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors.
In:
Human Gene Therapy. November 2016, 27(11).
(pp. A107-A108).
Mary Ann Liebert, Inc
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