Literature DB >> 35937482

Breakthrough News in Adenoviral Vector-Mediated AADC Gene Therapy: Lessons from the Success in AADC Deficiency and Possible Future Applications.

Maria João Malaquias1,2, Francesca Magrinelli1, Kailash P Bhatia1.   

Abstract

Entities:  

Keywords:  AADC deficiency; DDC; aromatic L‐amino acid decarboxylase; gene therapy; genetics

Year:  2022        PMID： 35937482      PMCID： PMC9346230          DOI： 10.1002/mdc3.13503

Source DB:  PubMed          Journal:  Mov Disord Clin Pract        ISSN： 2330-1619


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  5 in total

1.  Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach.

Authors:  K S Bankiewicz; J L Eberling; M Kohutnicka; W Jagust; P Pivirotto; J Bringas; J Cunningham; T F Budinger; J Harvey-White
Journal:  Exp Neurol       Date:  2000-07       Impact factor: 5.330

Review 2.  Aromatic amino acid decarboxylase deficiency: Molecular and metabolic basis and therapeutic outlook.

Authors:  Nastassja Himmelreich; Riccardo Montioli; Mariarita Bertoldi; Carla Carducci; Vincenzo Leuzzi; Corinne Gemperle; Todd Berner; Keith Hyland; Beat Thöny; Georg F Hoffmann; Carla B Voltattorni; Nenad Blau
Journal:  Mol Genet Metab       Date:  2019-03-27       Impact factor: 4.797

3.  Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial.

Authors:  Chadwick W Christine; R Mark Richardson; Amber D Van Laar; Marin E Thompson; Elisabeth M Fine; Omar S Khwaja; Chunming Li; Grace S Liang; Andreas Meier; Eiry W Roberts; Madeline L Pfau; Josh R Rodman; Krystof S Bankiewicz; Paul S Larson
Journal:  Neurology       Date:  2021-10-14       Impact factor: 11.800

4.  Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.

Authors:  Toni S Pearson; Nalin Gupta; Waldy San Sebastian; Jill Imamura-Ching; Amy Viehoever; Ana Grijalvo-Perez; Alex J Fay; Neha Seth; Shannon M Lundy; Youngho Seo; Miguel Pampaloni; Keith Hyland; Erin Smith; Gardenia de Oliveira Barbosa; Jill C Heathcock; Amy Minnema; Russell Lonser; J Bradley Elder; Jeffrey Leonard; Paul Larson; Krystof S Bankiewicz
Journal:  Nat Commun       Date:  2021-07-12       Impact factor: 14.919

5.  Gene therapy in the putamen for curing AADC deficiency and Parkinson's disease.

Authors:  Paul Wuh-Liang Hwu; Karl Kiening; Irina Anselm; David R Compton; Takeshi Nakajima; Thomas Opladen; Phillip L Pearl; Agathe Roubertie; Thomas Roujeau; Shin-Ichi Muramatsu
Journal:  EMBO Mol Med       Date:  2021-08-23       Impact factor: 12.137
  5 in total

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