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Orchard Therapeutics (United Kingdom)
Homepage:
orchard-tx.com
Alternate names:
Orchard Therapeutics (Europe),
Orchard Therapeutics Europe Ltd,
Orchard Therapeutics Limited,
Orchard Therapeutics plc
ROR:
050adjk75
OpenAlex ID:
I4405272939
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access
2022
·
Francesca Fumagalli
, Valeria Calbi
, et al.
·
The Lancet
·
317
Lentiviral gene therapy for X-linked chronic granulomatous disease
2020
·
Donald B. Kohn
, Claire Booth
, et al.
·
Nature Medicine
·
263
Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study
2019
·
Francesca Ferrua
, Maria Pia Cicalese
, et al.
·
The Lancet Haematology
·
225
PDF
Cell competition acts as a purifying selection to eliminate cells with mitochondrial defects during early mouse development
2021
·
Ana Lima
, Gabriele Lubatti
, et al.
·
Nature Metabolism
·
88
PDF
PYM50028, a novel, orally active, nonpeptide neurotrophic factor inducer, prevents and reverses neuronal damage induced by MPP + in mesencephalic neurons and by MPTP in a mouse model of Parkinson's disease
2008
·
Naomi P. Visanji
, Antonia Orsi
, et al.
·
The FASEB Journal
·
80
Young people's experiences of individual psychoanalytic psychotherapy
2006
·
Catherine Bury
, Hitesh Raval
, et al.
·
Psychology and Psychotherapy Theory Research and Practice
·
54
Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA
2019
·
Stuart M. Ellison
, Aiyin Liao
, et al.
·
Molecular Therapy — Methods & Clinical Development
·
50
PDF
IL1RAP expression and the enrichment of IL ‐33 activation signatures in severe neutrophilic asthma
2022
·
Yusef Eamon Badi
, Barbora Salcman
, et al.
·
Allergy
·
44
PDF
Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy
2025
·
Francesca Fumagalli
, Valeria Calbi
, et al.
·
New England Journal of Medicine
·
42
An international study of caregiver-reported burden and quality of life in metachromatic leukodystrophy
2022
·
Caroline Sevin
, Magalie Barth
, et al.
·
Orphanet Journal of Rare Diseases
·
37
PDF
The different ways to chitosan/hyaluronic acid nanoparticles: templated vs direct complexation. Influence of particle preparation on morphology, cell uptake and silencing efficiency
2019
·
Arianna Gennari
, Julio M Rios de la Rosa
, et al.
·
Beilstein Journal of Nanotechnology
·
36
PDF
Newborn Screening and Presymptomatic Treatment of Metachromatic Leukodystrophy
2024
·
Lucia Laugwitz
, Thomas P. Mechtler
, et al.
·
New England Journal of Medicine
·
32
The transformative potential of HSC gene therapy as a genetic medicine
2021
·
Pervinder Sagoo
, H. Bobby Gaspar
·
Gene Therapy
·
27
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier
2022
·
Bethan J. Critchley
, H. Bobby Gaspar
, et al.
·
Molecular Therapy
·
25
Understanding caregiver descriptions of initial signs and symptoms to improve diagnosis of metachromatic leukodystrophy
2022
·
F. Eichler
, Caroline Sevin
, et al.
·
Orphanet Journal of Rare Diseases
·
21
Distribution in the brain and possible neuroprotective effects of intranasally delivered multi-walled carbon nanotubes
2020
·
Marzia Soligo
, Fausto Maria Felsani
, et al.
·
Nanoscale Advances
·
19
Lentiviral gene therapy rescues p47phox chronic granulomatous disease and the ability to fight Salmonella infection in mice
2020
·
Andrea Schejtman
, Walmir Cutrim Aragão-Filho
, et al.
·
Gene Therapy
·
19
PDF
Lentiviral Gene Transfer Corrects Immune Abnormalities in XIAP Deficiency
2022
·
Joseph Topal
, Neelam Panchal
, et al.
·
Journal of Clinical Immunology
·
17
PDF
Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease
2020
·
Giuseppa Piras
, Claudia Montiel-Equihua
, et al.
·
Molecular Therapy — Methods & Clinical Development
·
17
PDF
A Nonhuman Primate PET Study: Measurement of Brain PDE4 Occupancy by Roflumilast Using (R)-[11C]Rolipram
2018
·
Akihiro Takano
, Tolga Uz
, et al.
·
Molecular Imaging and Biology
·
17
PDF
Long-Term Immune Recovery After Hematopoietic Stem Cell Transplantation for ADA Deficiency: a Single-Center Experience
2021
·
Alexandra Y. Kreins
, Helena F. Velasco
, et al.
·
Journal of Clinical Immunology
·
16
PDF
Higher precision, first tier newborn screening for metachromatic leukodystrophy using 16:1-OH-sulfatide
2024
·
Soumeya Bekri
, Annette Bley
, et al.
·
Molecular Genetics and Metabolism
·
15
A systematic review of clinical effectiveness and safety for historical and current treatment options for metachromatic leukodystrophy in children, including atidarsagene autotemcel
2023
·
Nigel Armstrong
, Andrew Olaye
, et al.
·
Orphanet Journal of Rare Diseases
·
15
PDF
Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort
2019
·
Donald B. Kohn
, Kit L. Shaw
, et al.
·
Blood
·
15
Water-soluble microgels made by radical polymerization in solution
2001
·
J. H. de Groot
, A. Zurutuza
, et al.
·
Colloid & Polymer Science
·
13
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Works count:
118
Citation count:
3,432
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