Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial.

By M. Kaplitt, A. Feigin, Chengke Tang, Helen L. Fitzsimons, P. Mattis, P. Lawlor, R. Bland, D. Young, Kristin Strybing, D. Eidelberg and 1 more. The Lancet, 2007

1,071 citations recorded by the source corpus.

Subfields: Genetics, Neurology

View Paper: https://www.semanticscholar.org/paper/0c36bc92fd8c13b3709ffc75737091238f056f8b

How researchers see this paper

The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.

In Genetics

1. Charles R. Gerfen

In Neurology

1. A. Schapira
2. Jose A. Obeso
3. Charles R. Gerfen
4. M. Farrer

Citations in the corpus

• Linking Molecules to Mood: New Insight Into the Biology of Depression (2010, neutral): “While we possess the technology to deliver specific genes into human brain through viral vectors (74), the beneficial effects of BDNF would have to outweigh potential negative effects on lowering seizure thresholds, altering indices of learning and memory as well as increasing the likelihood of mali”
• Role of ROS and RNS Sources in Physiological and Pathological Conditions (2016, background): “a small number of PD patients by improving motor performance [531, 532].”
• Current Clinical Applications of In Vivo Gene Therapy with AAVs (2020, background): “There were no treatment-related AEs, and all subjects demonstrated improvements in motor functioning with diminished thalamic metabolisms within the treated hemisphere.(46) In the phase 2 randomized controlled trial, patients with advanced PD received either a sham procedure (n = 23) or bilateral in”
• Immune responses to AAV vectors: overcoming barriers to successful gene therapy. (2013, neutral): “Conversely, the presence of anti-AAV antibodies does not seem to affect transduction efficiency when the vector is injected intraparenchymally or into the eye.(5-7,13,17,18,26,45-52)”
• The molecular neurobiology of depression (2008, neutral): “Researchers must better understand the biological basis for the efficacy of deep brain stimulation in depression, and must explore the therapeutic possibilities of viral-mediated gene delivery, which is being applied successfully to other neuropsychiatric disorder”
• Missing pieces in the Parkinson's disease puzzle (2010, methodology): “A second approach uses AAV2 to deliver glutamic acid decarboxylase (GAD) to the subthalamic nucleu”
• A designer AAV variant permits efficient retrograde access to projection neurons (2016, background): “Recombinant adeno-associated viruses (rAAVs) have emerged as an effective platform for in vivo gene therapy, as they mediate high-level transgene expression, are non-toxic, and evoke minimal immune responses (Kaplitt et al., 2007).”