Carlos J. Miranda

EIA University — Colombia

Medicine · Health Sciences

22h-index6.0kcitations49works2.02yr avg
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Research Topics

Amyotrophic Lateral Sclerosis Research(9), Neurogenetic and Muscular Disorders Research(9), CRISPR and Genetic Engineering(8), Mitochondrial Function and Pathology(7), Genetic Neurodegenerative Diseases(7)

Publications49 total

Intrathalamic delivery of adeno-associated viral vector expressing progranulin as gene therapy for GRN-related frontotemporal dementia
Molecular Therapy — Methods & Clinical Development·2025Open Access
MyoD is essential in rhabdomyosarcoma by promoting survival through differentiation and CYLD
iScience·2025· 2 citedOpen Access
FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1
Molecular Therapy·2025· 6 citedOpen Access
Pre‐clinical development of AVB‐101, an AAV gene therapy treatment for frontotemporal dementia with progranulin mutations
Alzheimer s & Dementia·2024Open Access
Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates
Molecular Therapy·2023· 22 citedOpen Access
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
Gene Therapy·2023· 36 citedOpen Access
FLT201: An AAV-mediated gene therapy for type 1 Gaucher disease designed to target difficult to reach tissues
Molecular Genetics and Metabolism·2021· 3 cited
Generation of β-Glucocerebrosidase variants with increased half-life in human plasma for liver directed AAV gene therapy aimed at the treatment of Gaucher disease type 1
Molecular Genetics and Metabolism·2021· 2 cited
One-off liver directed AAV gene therapy achieves long term uptake of acid beta-glucocerebrosidase by macrophages of affected tissues in Gaucher disease
Molecular Genetics and Metabolism·2020
Sox11 is an Activity-Regulated Gene with Dentate-Gyrus-Specific Expression Upon General Neural Activation
Cerebral Cortex·2019· 8 citedOpen Access
#065 Displasia Fibrosa Craniofacial‑ A propósito de um Caso Clínico
Revista Portuguesa de Estomatologia Medicina Dentária e Cirurgia Maxilofacial·2019Open Access
#023 Osteoma periférico solitário da mandíbula – Caso Clínico
Revista Portuguesa de Estomatologia Medicina Dentária e Cirurgia Maxilofacial·2019Open Access
Liver-Directed AAV Gene Therapy for Gaucher Disease
Blood·2019· 5 cited
Rett syndrome gene therapy improves survival and ameliorates behavioral phenotypes in MeCP2 null (S51.002)
Neurology·2019· 6 cited
Liver directed AAV gene therapy to treat Gaucher disease
Molecular Genetics and Metabolism·2019· 1 cited
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
New England Journal of Medicine·2017· 2.2k citedOpen Access
HSPB1 mutations causing hereditary neuropathy in humans disrupt non-cell autonomous protection of motor neurons
Experimental Neurology·2017· 21 citedOpen Access
NEUROD1 Instructs Neuronal Conversion in Non-Reactive Astrocytes
Stem Cell Reports·2017· 107 citedOpen Access
Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism
Proceedings of the National Academy of Sciences·2016· 180 citedOpen Access
480. Gene Therapy for Spinal Muscular Atrophy Type 1 Shows Potential to Improve Survival and Motor Functional Outcomes
Molecular Therapy·2016· 21 citedOpen Access
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Frequent Co-authors

Brian K. Kaspar(21), Kathrin Meyer(12), Lyndsey Braun(8), Miguel M. Santos(8), Shibi Likhite(7), Kevin D. Foust(7), Rose Sheridan(7), Romuald Corbau(7), SungWon Song(6), Massimo Pandolfo(6), Elisa Chisari(6), Jorge Sequeiros(5), Clément Cocita(5), Miriam Canavese(5), Jalpa Pandya(5), Amit C. Nathwani(5), Arthur H.M. Burghes(4), Mark E. Hester(4), Kar Men Mah(4), Laura Ferraiuolo(4)