Results 81 to 90 of about 784,273 (205)

Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease

, 2016
Enzyme replacement therapy (ERT) is the standard of care for several lysosomal storage diseases (LSDs). ERT, however, requires multiple and costly administrations and has limited efficacy.
Nusco, Edoardo, AURICCHIO, ALBERTO, Ferla, Rita, DE LEONIBUS, CHIARA, Alliegro, Marialuisa, SETTEMBRE, CARMINE   +5 more
core   +1 more source

Downregulation of Mannose-6-Phosphate Receptors in Fabry Disease Cardiomyopathy: A Potential Target for Enzyme Therapy Enhancement. [PDF]

J Clin Med, 2022
Frustaci A, Verardo R, Scialla R, Bagnato G, Verardo M, Alfarano M, Russo MA.   +6 more
europepmc   +1 more source

Bleeding Risk With Combination Intrapleural Fibrinolytic and Enzyme Therapy in Pleural Infection: An International, Multicenter, Retrospective Cohort Study. [PDF]

Chest, 2022
Akulian J, Bedawi EO, Abbas H, Argento C, Arnold DT, Balwan A, Batra H, Uribe Becerra JP, Belanger A, Berger K, Burks AC, Chang J, Chrissian AA, DiBardino DM, Fuentes XF, Gesthalter YB, Gilbert CR, Glisinski K, Godfrey M, Gorden JA, Grosu H, Gupta M, Kheir F, Ma KC, Majid A, Maldonado F, Maskell NA, Mehta H, Mercer J, Mullon J, Nelson D, Nguyen E, Pickering EM, Puchalski J, Reddy C, Revelo AE, Roller L, Sachdeva A, Sanchez T, Sathyanarayan P, Semaan R, Senitko M, Shojaee S, Story R, Thiboutot J, Wahidi M, Wilshire CL, Yu D, Zouk A, Rahman NM, Yarmus L, Interventional Pulmonary Outcomes Group.   +51 more
europepmc   +1 more source

Use of a direct, positive selection strategy to generate improved prodrug-activating enzymes for cancer gene therapy [PDF]

, 2011
E. coli NfsB nitroreductase (NTR) is currently being studied in combination with the prodrug CB1954, as a gene directed enzyme prodrug therapy. NTR reduces CB1954 at either the 2- or 4-nitro groups to produce highly cytotoxic hydroxylamine derivatives ...
Baker, Shelley Louise
core  

Does angiotensin-1 converting enzyme genotype influence motor or cognitive development after pre-term birth?

, 2005
Background Raised activity of the renin-angiotensin system (RAS) may both amplify inflammatory and free radical responses and decrease tissue metabolic efficiency and thus enhance cerebral injury in the preterm infant.
Montgomery, H, Harding, DR, Devadason David, Humphries, S, Whitelaw, A, David Davedason, Marlow Neil, Montgomery Hugh E, Marlow, N, Hugh E Montgomery, Davedason, D, Andrew Whitelaw, Humphries, SE, Neil Marlow, Montgomery, HE, Whitelaw Andrew, Humphries Steve E, Dhamrait Sukhbir, Devadason, D, Dhamrait, S, Steve E Humphries, Harding David R, David R Harding, Sukhbir Dhamrait   +23 more
core   +1 more source

Incremental biomarker and clinical outcomes after switch from enzyme therapy to eliglustat substrate reduction therapy in Gaucher disease. [PDF]

Mol Genet Metab Rep, 2021
Kleytman N, Ruan J, Ruan A, Zhang B, Murugesan V, Lin H, Guo L, Klinger K, Mistry PK.   +8 more
europepmc   +1 more source

Adequate hemodialysis does not compromise the cardioprotective effect of agalsidase alfa on patients with Fabry disease: a case report

Journal of Medical Case Reports
Background Fabry disease is an inherited lysosomal storage disease that can be reversed, or the progression slowed, by enzyme replacement therapy in the early stage.
Xiansen Wei, Shimin Jiang, Li Zhuo, Hong Jiang, Wenge Li   +4 more
doaj   +1 more source

Changing Times for CLN2 Disease: The Era of Enzyme Replacement Therapy

, 2020
Nicola Specchio, Nicola Pietrafusa, Marina Trivisano Rare and Complex Epilepsy Unit, Department of Neuroscience, Bambino Gesù Children’s Hospital, IRCCS, Rome, ItalyCorrespondence: Nicola SpecchioDepartment of Neuroscience, Bambino Ges ...
Pietrafusa N, Trivisano M, Specchio N
core  

Enzyme therapy for Pompe disease:from science to industrial enterprise

, 2002
Pompe disease or glycogen storage disease type II (OMIM 232300) is a metabolic myopathy with a broad clinical spectrum. Generalised muscle weakness combined with cardiomegaly presents within the first 3 months after birth, if the lysosomal alpha ...
Bijvoet, Agnes G.A., van der Ploeg, Ans T., Verbeet, Martin P., van den Hout, Hannerieke, Kroos, Marian A., Reuser, Arnold J.J.   +5 more
core   +1 more source

The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts

, 2009
In spite of the progress in the treatment of lysosomal storage diseases (LSDs), in some of these disorders the available therapies show limited efficacy and a need exists to identify novel therapeutic strategies.
Tarallo A, BARONE, MARIA VITTORIA, Cardone M, Porto C, Fontana F, PARENTI, GIANCARLO, Rossi B, Tuzzi MR, ANDRIA, GENEROSO   +8 more
core