Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
…, MP Cicalese, M Casiraghi, JJ Boelens, U Del Carro… - Science, 2013 - science.org
Introduction Metachromatic leukodystrophy (MLD) is a neurodegenerative lysosomal
storage disease caused by arylsulfatase A (ARSA) deficiency. The disease primarily affects …
storage disease caused by arylsulfatase A (ARSA) deficiency. The disease primarily affects …
Injection of adult neurospheres induces recovery in a chronic model of multiple sclerosis
… Ubaldo Del Carro … Data were compared using the Student's t-test for unpaired data, the
Mann-Whitney U-test for non-parametric data, or the χ 2 test. … Ubaldo Del Carro …
Mann-Whitney U-test for non-parametric data, or the χ 2 test. … Ubaldo Del Carro …
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial
…, G Antonioli, A Assanelli, MP Cicalese, U Del Carro… - The Lancet, 2016 - thelancet.com
Background Metachromatic leukodystrophy (a deficiency of arylsulfatase A [ARSA]) is a fatal
demyelinating lysosomal disease with no approved treatment. We aimed to assess the long-…
demyelinating lysosomal disease with no approved treatment. We aimed to assess the long-…
Guillain-Barré syndrome and COVID-19: an observational multicentre study from two Italian hotspot regions
…, F Castellani, G Zara, F Bianchi, U Del Carro… - Journal of Neurology …, 2021 - jnnp.bmj.com
Objective Single cases and small series of Guillain-Barré syndrome (GBS) have been
reported during the SARS-CoV-2 outbreak worldwide. We evaluated incidence and clinical …
reported during the SARS-CoV-2 outbreak worldwide. We evaluated incidence and clinical …
[HTML][HTML] Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase …
…, F Ferrua, M Migliavacca, F Tucci, V Gallo, U Del Carro… - The Lancet, 2022 - thelancet.com
Background Effective treatment for metachromatic leukodystrophy (MLD) remains a substantial
unmet medical need. In this study we investigated the safety and efficacy of atidarsagene …
unmet medical need. In this study we investigated the safety and efficacy of atidarsagene …
[HTML][HTML] Electrospun micro-and nanofiber tubes for functional nervous regeneration in sciatic nerve transections
S Panseri, C Cunha, J Lowery, U Del Carro… - BMC …, 2008 - Springer
Background Although many nerve prostheses have been proposed in recent years, in the
case of consistent loss of nervous tissue peripheral nerve injury is still a traumatic pathology …
case of consistent loss of nervous tissue peripheral nerve injury is still a traumatic pathology …
[HTML][HTML] Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells
…, M De Palma, A Quattrini, U Del Carro… - The Journal of …, 2004 - Am Soc Clin Investig
Gene-based delivery can establish a sustained supply of therapeutic proteins within the
nervous system. For diseases characterized by extensive CNS and peripheral nervous system (…
nervous system. For diseases characterized by extensive CNS and peripheral nervous system (…
[HTML][HTML] Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice
…, A Capotondo, S Fasano, U Del Carro… - The Journal of …, 2006 - Am Soc Clin Investig
Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by deficiency
of the enzyme arylsulfatase A (ARSA). MLD is characterized by progressive demyelination …
of the enzyme arylsulfatase A (ARSA). MLD is characterized by progressive demyelination …
[HTML][HTML] Ablation of the UPR-mediator CHOP restores motor function and reduces demyelination in Charcot-Marie-Tooth 1B mice
Deletion of serine 63 from P0 glycoprotein (P0S63del) causes Charcot-Marie-Tooth 1B
neuropathy in humans, and P0S63del produces a similar demyelinating neuropathy in …
neuropathy in humans, and P0S63del produces a similar demyelinating neuropathy in …
Human neural stem cells ameliorate autoimmune encephalomyelitis in non‐human primates
…, G Borsellino, C Cossetti, U Del Carro… - Annals of Neurology …, 2009 - Wiley Online Library
Objective Transplanted neural stem/precursor cells (NPCs) display peculiar therapeutic
plasticity in vivo. Although the replacement of cells was first expected as the prime therapeutic …
plasticity in vivo. Although the replacement of cells was first expected as the prime therapeutic …