Dysregulated Wnt and NFAT signaling in a Parkinson’s disease LRRK2 G2019S knock-in model

Authors: Andrea Wetzel, Si Hang Lei, Tiansheng Liu, Michael P. Hughes, Yunan Peng, Tristan McKay, Simon N. Waddington, Simone Grannò, Ahad A. Rahim, Kirsten Harvey

Source: Scientific Reports, Vol 14, Iss 1, Pp 1-22 (2024)

Subject Terms: LRRK2, Wnt signaling, NFAT signaling, Parkinson’s diseases, Immune system, LRRK2 G2019S, Medicine, Science

File Description: electronic resource

Relation: https://doaj.org/toc/2045-2322

Access URL: https://doaj.org/article/1938be2aee344346933815a42dfb5c2e

Macrophage Inhibitor Clodronate Enhances Liver Transduction of Lentiviral but Not Adeno-Associated Viral Vectors or mRNA Lipid Nanoparticles in Neonatal and Juvenile Mice

Authors: Loukia Touramanidou, Sonam Gurung, Claudiu A. Cozmescu, Dany Perocheau, Dale Moulding, Patrick F. Finn, Andrea Frassetto, Simon N. Waddington, Paul Gissen, Julien Baruteau

Source: Cells, Vol 13, Iss 23, p 1979 (2024)

Subject Terms: gene therapy, AAV, LNP mRNA, lentiviral vector, LV, macrophages, Cytology, QH573-671

File Description: electronic resource

Relation: https://www.mdpi.com/2073-4409/13/23/1979; https://doaj.org/toc/2073-4409

Access URL: https://doaj.org/article/013dd91f64db4e359881d03982e919bc

Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys

Authors: Julien Baruteau, Sharon C. Cunningham, Berna Seker Yilmaz, Dany P. Perocheau, Simon Eaglestone, Derek Burke, Adrian J. Thrasher, Simon N. Waddington, Leszek Lisowski, Ian E. Alexander, Paul Gissen

Source: Molecular Therapy: Methods & Clinical Development, Vol 23, Iss , Pp 135-146 (2021)

Subject Terms: adeno-associated virus, ornithine transcarbamylase deficiency, engineered capsid, biodistribution, liver, AAV, Genetics, QH426-470, Cytology, QH573-671

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S232905012100142X; https://doaj.org/toc/2329-0501

Access URL: https://doaj.org/article/10be24db36dd4d05b36ea1c89d212f01

Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation

Authors: John R. Counsell, Guillaume De Brabandere, Rajvinder Karda, Marc Moore, Antonio Greco, Alysha Bray, Juan Antinao Diaz, Dany P. Perocheau, Ulrike Mock, Simon N. Waddington

Source: Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 357-365 (2021)

Subject Terms: Genetics, QH426-470, Cytology, QH573-671

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050120302539; https://doaj.org/toc/2329-0501

Access URL: https://doaj.org/article/91dfe80765324ae6b172bc6c3bfd05e5

Dopamine Transporter Deficiency Syndrome (DTDS): Expanding the Clinical Phenotype and Precision Medicine Approaches

Authors: Joanne Ng, Serena Barral, Simon N. Waddington, Manju A. Kurian

Source: Cells, Vol 12, Iss 13, p 1737 (2023)

Subject Terms: dopamine transporter deficiency syndrome, infantile parkinsonism-dystonia, neurotransmitter, gene therapy, SLC6A3, DAT, Cytology, QH573-671

File Description: electronic resource

Relation: https://www.mdpi.com/2073-4409/12/13/1737; https://doaj.org/toc/2073-4409

Access URL: https://doaj.org/article/13b27937304742a694aef2d5149af478

Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice

Authors: John R. Counsell, Rajvinder Karda, Juan Antinao Diaz, Louise Carey, Tatiana Wiktorowicz, Suzanne M.K. Buckley, Shima Ameri, Joanne Ng, Julien Baruteau, Filipa Almeida, Rohan de Silva, Roberto Simone, Eleonora Lugarà, Gabriele Lignani, Dirk Lindemann, Axel Rethwilm, Ahad A. Rahim, Simon N. Waddington, Steven J. Howe

Source: Molecular Therapy: Nucleic Acids, Vol 12, Iss , Pp 626-634 (2018)

Subject Terms: Therapeutics. Pharmacology, RM1-950

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S216225311830146X; https://doaj.org/toc/2162-2531

Access URL: https://doaj.org/article/6238edf25cf544478737c499f40dbd36

Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer

Authors: Julien Baruteau, Dany P. Perocheau, Joanna Hanley, Maëlle Lorvellec, Eridan Rocha-Ferreira, Rajvinder Karda, Joanne Ng, Natalie Suff, Juan Antinao Diaz, Ahad A. Rahim, Michael P. Hughes, Blerida Banushi, Helen Prunty, Mariya Hristova, Deborah A. Ridout, Alex Virasami, Simon Heales, Stewen J. Howe, Suzanne M. K. Buckley, Philippa B. Mills, Paul Gissen, Simon N. Waddington

Source: Nature Communications, Vol 9, Iss 1, Pp 1-14 (2018)

Subject Terms: Science

File Description: electronic resource

Relation: https://doaj.org/toc/2041-1723

Access URL: https://doaj.org/article/bccd48b998f7466b8b4e37f6409acdb2

Continual conscious bioluminescent imaging in freely moving somatotransgenic mice

Authors: Rajvinder Karda, Dany P. Perocheau, Natalie Suff, Joanne Ng, Juliette M. K. M. Delhove, Suzanne M. K. Buckley, Samantha Richards, John R. Counsell, Henrik Hagberg, Mark R. Johnson, Tristan R. McKay, Simon N. Waddington

Source: Scientific Reports, Vol 7, Iss 1, Pp 1-13 (2017)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: https://doaj.org/toc/2045-2322

Access URL: https://doaj.org/article/9f6f64e65ad641139897de92958624f7

Cervical Gene Delivery of the Antimicrobial Peptide, Human β-Defensin (HBD)-3, in a Mouse Model of Ascending Infection-Related Preterm Birth

Authors: Natalie Suff, Rajvinder Karda, Juan Antinao Diaz, Joanne Ng, Julien Baruteau, Dany Perocheau, Peter W. Taylor, Dagmar Alber, Suzanne M. K. Buckley, Mona Bajaj-Elliott, Simon N. Waddington, Donald Peebles

Source: Frontiers in Immunology, Vol 11 (2020)

Subject Terms: cervix, gene therapy, preterm birth, antimicrobial peptides, ascending infection, Immunologic diseases. Allergy, RC581-607

File Description: electronic resource

Relation: https://www.frontiersin.org/article/10.3389/fimmu.2020.00106/full; https://doaj.org/toc/1664-3224

Access URL: https://doaj.org/article/87c4c20edc5c4f4ab1b9aa0b4309858a

In Vitro and In Vivo Evaluation of Human Adenovirus Type 49 as a Vector for Therapeutic Applications

Authors: Emily A. Bates, John R. Counsell, Sophie Alizert, Alexander T. Baker, Natalie Suff, Ashley Boyle, Angela C. Bradshaw, Simon N. Waddington, Stuart A. Nicklin, Andrew H. Baker, Alan L. Parker

Source: Viruses, Vol 13, Iss 8, p 1483 (2021)

Subject Terms: adenovirus, viral vector, gene therapy, vaccines, Microbiology, QR1-502

File Description: electronic resource

Relation: https://www.mdpi.com/1999-4915/13/8/1483; https://doaj.org/toc/1999-4915

Access URL: https://doaj.org/article/b185ff6201cc438789403464eeb6d6d7

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development

Authors: Giulia Massaro, Amy F. Geard, Wenfei Liu, Oliver Coombe-Tennant, Simon N. Waddington, Julien Baruteau, Paul Gissen, Ahad A. Rahim

Source: Biomolecules, Vol 11, Iss 4, p 611 (2021)

Subject Terms: lysosomal diseases, gene therapy, viral vectors, Microbiology, QR1-502

File Description: electronic resource

Relation: https://www.mdpi.com/2218-273X/11/4/611; https://doaj.org/toc/2218-273X

Access URL: https://doaj.org/article/0d64169604c944538a096cbb0b3f671b

Lentiviral vectors can be used for full-length dystrophin gene therapy

Authors: John R. Counsell, Zeinab Asgarian, Jinhong Meng, Veronica Ferrer, Conrad A. Vink, Steven J. Howe, Simon N. Waddington, Adrian J. Thrasher, Francesco Muntoni, Jennifer E. Morgan, Olivier Danos

Source: Scientific Reports, Vol 7, Iss 1, Pp 1-10 (2017)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: https://doaj.org/toc/2045-2322

Access URL: https://doaj.org/article/a4e4bc5af0674a76abc106a806bbacbb

Regulation of post-Golgi LH3 trafficking is essential for collagen homeostasis

Authors: Blerida Banushi, Federico Forneris, Anna Straatman-Iwanowska, Adam Strange, Anne-Marie Lyne, Clare Rogerson, Jemima J. Burden, Wendy E. Heywood, Joanna Hanley, Ivan Doykov, Kornelis R. Straatman, Holly Smith, Danai Bem, Janos Kriston-Vizi, Gema Ariceta, Maija Risteli, Chunguang Wang, Rosalyn E. Ardill, Marcin Zaniew, Julita Latka-Grot, Simon N. Waddington, S. J. Howe, Francesco Ferraro, Asllan Gjinovci, Scott Lawrence, Mark Marsh, Mark Girolami, Laurent Bozec, Kevin Mills, Paul Gissen

Source: Nature Communications, Vol 7, Iss 1, Pp 1-14 (2016)

Subject Terms: Science

File Description: electronic resource

Relation: https://doaj.org/toc/2041-1723

Access URL: https://doaj.org/article/e77a5c9ad1f34f37a39287201a081ba7

NRF2 Orchestrates the Metabolic Shift during Induced Pluripotent Stem Cell Reprogramming

Authors: Kate E. Hawkins, Shona Joy, Juliette M.K.M. Delhove, Vassilios N. Kotiadis, Emilio Fernandez, Lorna M. Fitzpatrick, James R. Whiteford, Peter J. King, Juan P. Bolanos, Michael R. Duchen, Simon N. Waddington, Tristan R. McKay

Source: Cell Reports, Vol 14, Iss 8, Pp 1883-1891 (2016)

Subject Terms: Biology (General), QH301-705.5

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2211124716300705; https://doaj.org/toc/2211-1247

Access URL: https://doaj.org/article/272ceabc933b4f23acf31d95556d9320

Publisher Correction: In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques

Authors: Panicos Shangaris, Stavros P. Loukogeorgakis, Sindhu Subramaniam, Christina Flouri, Laurence H. Jackson, Wei Wang, Michael P. Blundell, Shanrun Liu, Simon Eaton, Nahla Bakhamis, Durrgah Latchumi Ramachandra, Panayiotis Maghsoudlou, Luca Urbani, Simon N. Waddington, Ayad Eddaoudi, Joy Archer, Michael N. Antoniou, Daniel J. Stuckey, Manfred Schmidt, Adrian J. Thrasher, Thomas M. Ryan, Paolo De Coppi, Anna L. David

Source: Scientific Reports, Vol 9, Iss 1, Pp 1-1 (2019)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: https://doaj.org/toc/2045-2322

Access URL: https://doaj.org/article/ad29baa02fe44e57b3f23000d2ac133d

Urea Cycle Related Amino Acids Measured in Dried Bloodspots Enable Long-Term In Vivo Monitoring and Therapeutic Adjustment

Authors: Julien Baruteau, Youssef Khalil, Stephanie Grunewald, Marta Zancolli, Anupam Chakrapani, Maureen Cleary, James Davison, Emma Footitt, Simon N. Waddington, Paul Gissen, Philippa Mills

Source: Metabolites, Vol 9, Iss 11, p 275 (2019)

Subject Terms: tandem mass spectrometry, dried blood spots, urea cycle, amino acids, argininosuccinate lyase, Microbiology, QR1-502

File Description: electronic resource

Relation: https://www.mdpi.com/2218-1989/9/11/275; https://doaj.org/toc/2218-1989

Access URL: https://doaj.org/article/b37464dd2746486582cd7f094e19969b

Evidence for Contribution of CD4+CD25+ Regulatory T Cells in Maintaining Immune Tolerance to Human Factor IX following Perinatal Adenovirus Vector Delivery

Authors: Megha S. Nivsarkar, Suzanne M. K. Buckley, Alan L. Parker, Dany Perocheau, Tristan R. McKay, Ahad A. Rahim, Steven J. Howe, Simon N. Waddington

Source: Journal of Immunology Research, Vol 2015 (2015)

Subject Terms: Immunologic diseases. Allergy, RC581-607

File Description: electronic resource

Relation: https://doaj.org/toc/2314-8861; https://doaj.org/toc/2314-7156

Access URL: https://doaj.org/article/ac77022689344402bb0548d5af363f32

Current therapies for the soluble lysosomal forms of neuronal ceroid lipofuscinosis.

Authors: Andrew M.S. Wong, Ahad A. Rahim, Simon N. Waddington, Jonathan D. Cooper

Source: Biochemical Society Transactions; Dec2010, Vol. 38 Issue 6, p1484-1488, 5p

Subject Terms: NEURONAL ceroid-lipofuscinosis, NEURODEGENERATION, GENETIC disorders, MEMBRANE proteins, ENDOCYTOSIS, GENE therapy, STEM cell treatment, THERAPEUTICS

In utero gene transfer to the mouse nervous system.

Authors: Ahad A. Rahim, Andrew M.S. Wong, Suzanne M.K. Buckley, Jerry K.Y. Chan, Anna L. David, Jonathan D. Cooper, Charles Coutelle, Donald M. Peebles, Simon N. Waddington

Source: Biochemical Society Transactions; Dec2010, Vol. 38 Issue 6, p1489-1493, 5p

Subject Terms: GENETIC transformation, NERVOUS system, FETUS, IMMUNE system, GENETIC disorder treatment, PATHOLOGICAL physiology, MUSCULAR atrophy, LABORATORY mice, THERAPEUTICS

The differentiation and engraftment potential of mouse hematopoietic stem cells is maintained after bio-electrospray.

Authors: Kerol Bartolovic, Napachanok Mongkoldhumrongkul, Simon N. Waddington, Suwan N. Jayasinghe, Steven J. Howe

Source: Analyst; Jan2010, Vol. 135 Issue 1, p157-164, 8p

Subject Terms: HEMATOPOIETIC stem cells, CELL differentiation, LABORATORY mice, BIOCHEMISTRY technique, CELL populations, BIOLOGICAL assay, BIOLOGICAL models