Mission possible: Gene therapy for inherited metabolic diseases
Fuente:
Wiley Open Access
Saved in:
| Main Authors: | Julien Baruteau, Nandaki Keshavan, Charles P. Venditti |
|---|---|
| Format: | Artículo Open Access |
| Published: |
Wiley
2024
|
| Subjects: | |
| Online Access: | |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
Similar Items
Liver‐directed gene therapy for inherited metabolic diseases
by: Julien Baruteau, et al.
Published: (2024)
by: Julien Baruteau, et al.
Published: (2024)
Gene therapy for mitochondrial disorders
by: Nandaki Keshavan, et al.
Published: (2024)
by: Nandaki Keshavan, et al.
Published: (2024)
First in Human Gene Editing for an Inherited Metabolic Disease
by: Shamima Rahman, et al.
Published: (2025)
by: Shamima Rahman, et al.
Published: (2025)
Cellular mechanisms of acute rhabdomyolysis in inherited metabolic diseases
by: Hortense de Calbiac, et al.
Published: (2024)
by: Hortense de Calbiac, et al.
Published: (2024)
What can pediatricians learn from adult inherited metabolic diseases?
by: Fanny Mochel
Published: (2024)
by: Fanny Mochel
Published: (2024)
Gene replacement therapies for inherited disorders of neurotransmission: Current progress in succinic semialdehyde dehydrogenase deficiency
by: Henry H. C. Lee, et al.
Published: (2024)
by: Henry H. C. Lee, et al.
Published: (2024)
Parental and child's psychosocial and financial burden living with an inherited metabolic disease identified by newborn screening
by: Elena Schnabel‐Besson, et al.
Published: (2024)
by: Elena Schnabel‐Besson, et al.
Published: (2024)
Delivering the Message: Translating mRNA Therapy for Liver Inherited Metabolic Diseases
by: Sonam Gurung, et al.
Published: (2025)
by: Sonam Gurung, et al.
Published: (2025)
Mouse models for inherited monoamine neurotransmitter disorders
by: Beat Thöny, et al.
Published: (2024)
by: Beat Thöny, et al.
Published: (2024)
Natural history of valve disease in patients with mucopolysaccharidosis II and the impact of enzyme replacement therapy
by: Christoph Kampmann, et al.
Published: (2024)
by: Christoph Kampmann, et al.
Published: (2024)
Human glyoxylate metabolism revisited: New insights pointing to multi‐organ involvement with implications for siRNA‐based therapies in primary hyperoxaluria
by: Ronald J. A. Wanders, et al.
Published: (2024)
by: Ronald J. A. Wanders, et al.
Published: (2024)
MRPS Genes Causing Leukoencephalopathy With Profound Cerebral Folate Deficiency in Adults
by: Daniele Mandia, et al.
Published: (2026)
by: Daniele Mandia, et al.
Published: (2026)
Current global vitamin and cofactor prescribing practices for primary mitochondrial diseases: Results of a European reference network survey
by: Julia Neugebauer, et al.
Published: (2024)
by: Julia Neugebauer, et al.
Published: (2024)
Gene Dosage Sensitivity and Human Genetic Diseases
by: Reiner A. Veitia, et al.
Published: (2025)
by: Reiner A. Veitia, et al.
Published: (2025)
A phase III, open‐label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme replacement therapies
by: Myrl Holida, et al.
Published: (2024)
by: Myrl Holida, et al.
Published: (2024)
Correction to “Comparative Analysis of Gene and Disease Selection in Genomic Newborn Screening Studies”
Published: (2025)
Published: (2025)
Clinical pharmacology considerations for first‐in‐human clinical trials for enzyme replacement therapy
by: Sydney Stern, et al.
Published: (2024)
by: Sydney Stern, et al.
Published: (2024)
Imaging readiness in the gene therapy era‐exploring standardized protocols for response assessment
by: Asthik Biswas, et al.
Published: (2024)
by: Asthik Biswas, et al.
Published: (2024)
Comparative analysis of gene and disease selection in genomic newborn screening studies
by: Isabel R. Betzler, et al.
Published: (2024)
by: Isabel R. Betzler, et al.
Published: (2024)
Long‐term cardiovascular outcomes and mortality with enzyme replacement therapy in patients with mucopolysaccharidosis type II
by: Ji Hee Kwak, et al.
Published: (2024)
by: Ji Hee Kwak, et al.
Published: (2024)
Liver‐Directed Gene Therapy Mitigates Early Nephropathy in Murine Glycogen Storage Disease Type Ia
by: Cheol Lee, et al.
Published: (2025)
by: Cheol Lee, et al.
Published: (2025)
The Metabolic Treatabolome and Inborn Errors of Metabolism Knowledgebase therapy tool: Do not miss the opportunity to treat!
by: Bibiche den Hollander, et al.
Published: (2025)
by: Bibiche den Hollander, et al.
Published: (2025)
Brain morphometry in hepatic Wilson disease patients
by: Parya Rahimi, et al.
Published: (2024)
by: Parya Rahimi, et al.
Published: (2024)
Unifying biology of neurodegeneration in lysosomal storage diseases
by: Anna M. Ludlaim, et al.
Published: (2025)
by: Anna M. Ludlaim, et al.
Published: (2025)
Neuronopathic Gaucher disease: Rare in the West, common in the East
by: Ozlem Goker‐Alpan, et al.
Published: (2024)
by: Ozlem Goker‐Alpan, et al.
Published: (2024)
Signal Peptide Engineering and Codon Optimization to Enhance α‐Gal A Activity for rAAV Gene Therapy of Fabry Disease
by: Siwu He, et al.
Published: (2026)
by: Siwu He, et al.
Published: (2026)
A 6‐month randomized controlled trial for vitamin E supplementation in pediatric patients with Gaucher disease: Effect on oxidative stress, disease severity and hepatic complications
by: Amira Abdel Moneam Adly, et al.
Published: (2024)
by: Amira Abdel Moneam Adly, et al.
Published: (2024)
Long term survival in patients with classic infantile Pompe disease reveals a spectrum with progressive brain abnormalities and changes in cognitive functioning
by: J. J. A. van den Dorpel, et al.
Published: (2024)
by: J. J. A. van den Dorpel, et al.
Published: (2024)
Natural disease course of chronic visceral acid sphingomyelinase deficiency in adults: A first step toward treatment criteria
by: Eline C. B. Eskes, et al.
Published: (2024)
by: Eline C. B. Eskes, et al.
Published: (2024)
Safety and Efficacy of DTX401 , an AAV8 ‐Mediated Liver‐Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a ( GSDIa )
by: David A. Weinstein, et al.
Published: (2025)
by: David A. Weinstein, et al.
Published: (2025)
Nontargeted urine metabolomic analysis of acute intermittent porphyria reveals novel interactions between bile acids and heme metabolism: New promising biomarkers for the long‐term management of patients
by: Thibaud Lefebvre, et al.
Published: (2024)
by: Thibaud Lefebvre, et al.
Published: (2024)
Restoring galactose metabolism without restoring GALT rescues both compromised survival in larvae and an adult climbing deficit in a GALT‐null D. Melanogaster model of classic galactosemia
by: Jennifer M. I. Daenzer, et al.
Published: (2024)
by: Jennifer M. I. Daenzer, et al.
Published: (2024)
Uncommon case of mitochondrial disease: Mild amyotrophy of the legs and symmetrical lipomatosis of the arms
by: Leslie Bercu, et al.
Published: (2024)
by: Leslie Bercu, et al.
Published: (2024)
Clinical and Biochemical Characterization of Fabry Disease Associated GLA Gene Variants: Data From a Large Cohort of 469 Thousand Genotyped Subjects of the UK Biobank Database
by: Antonina Giammanco, et al.
Published: (2025)
by: Antonina Giammanco, et al.
Published: (2025)
Epidemiology and economic burden of Wilson disease in France: A nationwide population‐based study
by: Shona Fang, et al.
Published: (2024)
by: Shona Fang, et al.
Published: (2024)
Executive and adaptive function impacts long‐term outcomes for adults with maple syrup urine disease
by: Jessica I. Gold, et al.
Published: (2024)
by: Jessica I. Gold, et al.
Published: (2024)
The PompeQoL questionnaire: Development and validation of a new measure for children and adolescents with Pompe disease
by: Moritz Ilan Truninger, et al.
Published: (2024)
by: Moritz Ilan Truninger, et al.
Published: (2024)
Regulatory news: Cipaglucosidase alfa‐atga (Pombiliti) coadministered with Miglustat (Opfolda) for adults with late‐onset Pompe disease
by: Daniela V. Luquetti, et al.
Published: (2024)
by: Daniela V. Luquetti, et al.
Published: (2024)
Integration of multi‐omics layers empowers precision diagnosis through unveiling pathogenic mechanisms on maple syrup urine disease
by: Juan Ramón Tejedor, et al.
Published: (2024)
by: Juan Ramón Tejedor, et al.
Published: (2024)
Repurposing empagliflozin in individuals with glycogen storage disease Ib: A value‐based healthcare approach and systematic benefit‐risk assessment
by: Terry G. J. Derks, et al.
Published: (2024)
by: Terry G. J. Derks, et al.
Published: (2024)
Similar Items
-
Liver‐directed gene therapy for inherited metabolic diseases
by: Julien Baruteau, et al.
Published: (2024) -
Gene therapy for mitochondrial disorders
by: Nandaki Keshavan, et al.
Published: (2024) -
First in Human Gene Editing for an Inherited Metabolic Disease
by: Shamima Rahman, et al.
Published: (2025) -
Cellular mechanisms of acute rhabdomyolysis in inherited metabolic diseases
by: Hortense de Calbiac, et al.
Published: (2024) -
What can pediatricians learn from adult inherited metabolic diseases?
by: Fanny Mochel
Published: (2024)