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Literature DB >> 27324445

Timing of Gene Therapy Interventions: The Earlier, the Better.

Steven J Gray¹.

Abstract

Year: 2016 PMID： 27324445 PMCID： PMC4923318 DOI： 10.1038/mt.2016.20

Source DB: PubMed Journal: Mol Ther ISSN： 1525-0016 Impact factor: 11.454

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15 in total

Review 1. Molecular genetics of mucopolysaccharidosis type IIIA and IIIB: Diagnostic, clinical, and biological implications.

Authors: G Yogalingam; J J Hopwood
Journal: Hum Mutat Date: 2001-10 Impact factor: 4.878

2. Newborn screening for Krabbe disease: the New York State model.

Authors: Patricia K Duffner; Michele Caggana; Joseph J Orsini; David A Wenger; Marc C Patterson; Carl J Crosley; Joanne Kurtzberg; Georgianne L Arnold; Maria L Escolar; Darius J Adams; Mary R Andriola; Alan M Aron; Emma Ciafaloni; Alexandra Djukic; Richard W Erbe; Patricia Galvin-Parton; Laura E Helton; Edwin H Kolodny; Barry E Kosofsky; David F Kronn; Jennifer M Kwon; Paul A Levy; Jill Miller-Horn; Thomas P Naidich; Joan E Pellegrino; James M Provenzale; Stanley J Rothman; Melissa P Wasserstein
Journal: Pediatr Neurol Date: 2009-04 Impact factor: 3.372

3. A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome).

Authors: M Bhaumik; V J Muller; T Rozaklis; L Johnson; K Dobrenis; R Bhattacharyya; S Wurzelmann; P Finamore; J J Hopwood; S U Walkley; P Stanley
Journal: Glycobiology Date: 1999-12 Impact factor: 4.313

4. Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer.

Authors: Albert Ruzo; Sara Marcó; Miquel García; Pilar Villacampa; Albert Ribera; Eduard Ayuso; Lucca Maggioni; Federico Mingozzi; Virginia Haurigot; Fatima Bosch
Journal: Hum Gene Ther Date: 2012-10-17 Impact factor: 5.695

5. Mucopolysaccharidosis IIIB, a lysosomal storage disease, triggers a pathogenic CNS autoimmune response.

Authors: Smruti Killedar; Julianne Dirosario; Erin Divers; Phillip G Popovich; Douglas M McCarty; Haiyan Fu
Journal: J Neuroinflammation Date: 2010-07-16 Impact factor: 8.322

6. Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy.

Authors: Virginia Haurigot; Sara Marcó; Albert Ribera; Miguel Garcia; Albert Ruzo; Pilar Villacampa; Eduard Ayuso; Sònia Añor; Anna Andaluz; Mercedes Pineda; Gemma García-Fructuoso; Maria Molas; Luca Maggioni; Sergio Muñoz; Sandra Motas; Jesús Ruberte; Federico Mingozzi; Martí Pumarola; Fatima Bosch
Journal: J Clin Invest Date: 2013-07-01 Impact factor: 14.808

7. A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice.

Authors: Seemin Seher Ahmed; Huapeng Li; Chunyan Cao; Elif M Sikoglu; Andrew R Denninger; Qin Su; Samuel Eaton; Ana A Liso Navarro; Jun Xie; Sylvia Szucs; Hongwei Zhang; Constance Moore; Daniel A Kirschner; Thomas N Seyfried; Terence R Flotte; Reuben Matalon; Guangping Gao
Journal: Mol Ther Date: 2013-07-02 Impact factor: 11.454

8. Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis.

Authors: Dolan Sondhi; Daniel A Peterson; Andrew M Edelstein; Katrina del Fierro; Neil R Hackett; Ronald G Crystal
Journal: Exp Neurol Date: 2008-04-30 Impact factor: 5.330

Review 9. Sanfilippo syndrome: a mini-review.

Authors: M J Valstar; G J G Ruijter; O P van Diggelen; B J Poorthuis; F A Wijburg
Journal: J Inherit Metab Dis Date: 2008-04-04 Impact factor: 4.982

10. Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease.

Authors: Mario A Cabrera-Salazar; Eric M Roskelley; Jie Bu; Bradley L Hodges; Nelson Yew; James C Dodge; Lamya S Shihabuddin; Istvan Sohar; David E Sleat; Ronald K Scheule; Beverly L Davidson; Seng H Cheng; Peter Lobel; Marco A Passini
Journal: Mol Ther Date: 2007-07-17 Impact factor: 11.454

6 in total

1. Engineered AAVs for non-invasive gene delivery to rodent and non-human primate nervous systems.

Authors: Xinhong Chen; Sripriya Ravindra Kumar; Cameron D Adams; Daping Yang; Tongtong Wang; Damien A Wolfe; Cynthia M Arokiaraj; Victoria Ngo; Lillian J Campos; Jessica A Griffiths; Takako Ichiki; Sarkis K Mazmanian; Peregrine B Osborne; Janet R Keast; Cory T Miller; Andrew S Fox; Isaac M Chiu; Viviana Gradinaru
Journal: Neuron Date: 2022-05-27 Impact factor: 18.688

Review 2. Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.

Authors: Carsten W Lederer; Lola Koniali; Tina Buerki-Thurnherr; Panayiota L Papasavva; Stefania La Grutta; Amelia Licari; Frantisek Staud; Donato Bonifazi; Marina Kleanthous
Journal: Pharmaceutics Date: 2022-04-05 Impact factor: 6.525

Review 6. Systemic cell therapy for muscular dystrophies : The ultimate transplantable muscle progenitor cell and current challenges for clinical efficacy.

Authors: Hans van Bokhoven; Derick G Wansink; C Rosanne M Ausems; Baziel G M van Engelen
Journal: Stem Cell Rev Rep Date: 2020-12-21 Impact factor: 5.739