2019 |
Prabhakar, S; Cheah, P S; Zhang, X; Zinter, M; Gianatasio, M; Hudry, E; Bronson, R T; Kwiatkowski, D J; Stemmer-Rachamimov, A; Maguire, C A; Sena-Esteves, M; Tannous, B A; Breakefield, X O Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1 Journal Article Molecular Therapy - Methods and Clinical Development, 15 , pp. 18-26, 2019, ISSN: 23290501, (cited By 2). Abstract | Links | BibTeX | Tags: Adeno Associated Virus, Adeno Associated Virus Vector, Animal Experiment, Animal Model, Article, Beta Actin, Blood Brain Barrier, Body Weight, Body Weight Gain, Brain Nerve Cell, Brain Ventricle, Cell Proliferation, Complementary DNA, Controlled Study, Cre Recombinase, Drug Efficacy, Female, Gene, Gene Replacement Therapy, Hamartin, HEK293 Cell Line, Hydrocephalus, Immunohistochemistry, Inverted Terminal Repeat, Long Term Care, Male, Motor Activity, Motor Performance, Mouse, Nonhuman, Priority Journal, Promoter Region, Protein Function, Protein Phosphorylation, Quantitative Analysis, Subventricular Zone, Survival Time, Tuberous Sclerosis, Tuberous Sclerosis Type 1, Vascularization, Viral Gene Delivery System @article{Prabhakar201918, title = {Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1}, author = {S Prabhakar and P S Cheah and X Zhang and M Zinter and M Gianatasio and E Hudry and R T Bronson and D J Kwiatkowski and A Stemmer-Rachamimov and C A Maguire and M Sena-Esteves and B A Tannous and X O Breakefield}, url = {https://www.scopus.com/inward/record.uri?eid=2-s2.0-85070908794&doi=10.1016%2fj.omtm.2019.08.003&partnerID=40&md5=b169187dde0d3b05f8a9d5295a4ad8c4}, doi = {10.1016/j.omtm.2019.08.003}, issn = {23290501}, year = {2019}, date = {2019-01-01}, journal = {Molecular Therapy - Methods and Clinical Development}, volume = {15}, pages = {18-26}, publisher = {Cell Press}, abstract = {Tuberous sclerosis complex (TSC) is a tumor suppressor syndrome caused by mutations in TSC1 or TSC2, encoding hamartin and tuberin, respectively. These proteins act as a complex that inhibits mammalian target of rapamycin (mTOR)-mediated cell growth and proliferation. Loss of either protein leads to overgrowth in many organs, including subependymal nodules, subependymal giant cell astrocytomas, and cortical tubers in the human brain. Neurological manifestations in TSC include intellectual disability, autism, hydrocephalus, and epilepsy. In a stochastic mouse model of TSC1 brain lesions, complete loss of Tsc1 is achieved in homozygous Tsc1-floxed mice in a subpopulation of neural cells in the brain by intracerebroventricular (i.c.v.) injection at birth of an adeno-associated virus (AAV) vector encoding Cre recombinase. This results in median survival of 38 days and brain pathology, including subependymal lesions and enlargement of neuronal cells. Remarkably, when these mice were injected intravenously on day 21 with an AAV9 vector encoding hamartin, most survived at least up to 429 days in apparently healthy condition with marked reduction in brain pathology. Thus, a single intravenous administration of an AAV vector encoding hamartin restored protein function in enough cells in the brain to extend lifespan in this TSC1 mouse model. © 2019}, note = {cited By 2}, keywords = {Adeno Associated Virus, Adeno Associated Virus Vector, Animal Experiment, Animal Model, Article, Beta Actin, Blood Brain Barrier, Body Weight, Body Weight Gain, Brain Nerve Cell, Brain Ventricle, Cell Proliferation, Complementary DNA, Controlled Study, Cre Recombinase, Drug Efficacy, Female, Gene, Gene Replacement Therapy, Hamartin, HEK293 Cell Line, Hydrocephalus, Immunohistochemistry, Inverted Terminal Repeat, Long Term Care, Male, Motor Activity, Motor Performance, Mouse, Nonhuman, Priority Journal, Promoter Region, Protein Function, Protein Phosphorylation, Quantitative Analysis, Subventricular Zone, Survival Time, Tuberous Sclerosis, Tuberous Sclerosis Type 1, Vascularization, Viral Gene Delivery System}, pubstate = {published}, tppubtype = {article} } Tuberous sclerosis complex (TSC) is a tumor suppressor syndrome caused by mutations in TSC1 or TSC2, encoding hamartin and tuberin, respectively. These proteins act as a complex that inhibits mammalian target of rapamycin (mTOR)-mediated cell growth and proliferation. Loss of either protein leads to overgrowth in many organs, including subependymal nodules, subependymal giant cell astrocytomas, and cortical tubers in the human brain. Neurological manifestations in TSC include intellectual disability, autism, hydrocephalus, and epilepsy. In a stochastic mouse model of TSC1 brain lesions, complete loss of Tsc1 is achieved in homozygous Tsc1-floxed mice in a subpopulation of neural cells in the brain by intracerebroventricular (i.c.v.) injection at birth of an adeno-associated virus (AAV) vector encoding Cre recombinase. This results in median survival of 38 days and brain pathology, including subependymal lesions and enlargement of neuronal cells. Remarkably, when these mice were injected intravenously on day 21 with an AAV9 vector encoding hamartin, most survived at least up to 429 days in apparently healthy condition with marked reduction in brain pathology. Thus, a single intravenous administration of an AAV vector encoding hamartin restored protein function in enough cells in the brain to extend lifespan in this TSC1 mouse model. © 2019 |
2010 |
Patel, V; Maj, M; Flisher, A J; Silva, De M J; Koschorke, M; Prince, M; Tempier, R; Riba, M B; Sanchez, M; Campodonico, F D; Risco, L; Gask, L; Wahlberg, H; Roca, M; Lecic-Tosevski, D; Soghoyan, A; Moussaoui, D; Baddoura, C; Adeyemi, J; Rataemane, S; Jalili, S A; Mohandas, E; Shinfuku, N; Freidin, J; Stagnaro, J C; Puig, I J; Kirkby, K; Musalek, M; Ismayilov, N; Rabbani, G; Harvey, S; Sabbe, B; Noya-Tapia, N; Burgic-Radmanovic, M; Hetem, L A; Vasconcellos, F; Maass, J; Miranda, C; Papaneophytou, N; Raboch, J; Fink-Jensen, A; Okasha, A; Korkeila, J; Guelfi, J D; Schneider, F; Ohene, S; Christodoulou, G; Soldatos, C R; Barrera, S K E Q; Mendoza, M; Kallivayalil, R A; Gudarzi, S S; Lafta, M R; Bassi, M; Clerici, M; Gibson, R; Kojima, T; Nurmagambetova, S; Cho, S -C; Kadyrova, T; Mikati, N; Bajraktarov, S; Yen, T H; Ayushjav, B; Stevovic, L I; Molina, J S S; Gureje, O; Johannessen, J O; Chaudhry, H R; Al-Ashhab, B; Araszkiewicz, A; Prelipceanu, D; Krasnov, V; Bogdanov, A; Jasovic-Gasic, M; Vavrusova, L; Pregelj, P; Liria, A F; Abdelrahman, A; Udomratn, P; Ulas, H; Gokaip, P; Kigozi, F N; Richardson, G Reducing the treatment gap for mental disorders: A WPA survey Journal Article World Psychiatry, 9 (3), pp. 169-176, 2010, ISSN: 17238617, (cited By 127). Abstract | Links | BibTeX | Tags: Anxiety Disorder, Article, Atomoxetine, Atypical Antipsychotic Agent, Autism, Benzodiazepine, Bipolar Disorder, Central Nervous System Stimulants, Cholinesterase Inhibitor, Cognitive Therapy, Community Mental Health Center, Conduct Disorder, Cost Effectiveness Analysis, Dementia, Depression, Evidence-based Practice, Family, Family Therapy, Haloperidol, Health Care, Health Care Access, Health Care Delivery, Health Care Personnel, Health Practitioner, Health Survey, Help Seeking Behavior, Home Mental Health Care, Human, Hyperkinesia, Long Term Care, Lowest Income Group, Mental Deficiency, Mental Disease, Mental Health, Mental Health Care, Mental Health Service, Nootropic Agent, Open Ended Questionnaire, Outcome Assessment, Patient Compliance, Personality Disorder, Practice Guideline, Priority Journal, Psychiatry, Psychoeducation, Psychotherapy, Schizophrenia, Serotonin Noradrenalin Reuptake Inhibitor, Serotonin Uptake Inhibitor, Substance Abuse, Therapy, Therapy Delay, Tricyclic Antidepressant Agent, World Health Organization @article{Patel2010169, title = {Reducing the treatment gap for mental disorders: A WPA survey}, author = {V Patel and M Maj and A J Flisher and M J De Silva and M Koschorke and M Prince and R Tempier and M B Riba and M Sanchez and F D Campodonico and L Risco and L Gask and H Wahlberg and M Roca and D Lecic-Tosevski and A Soghoyan and D Moussaoui and C Baddoura and J Adeyemi and S Rataemane and S A Jalili and E Mohandas and N Shinfuku and J Freidin and J C Stagnaro and I J Puig and K Kirkby and M Musalek and N Ismayilov and G Rabbani and S Harvey and B Sabbe and N Noya-Tapia and M Burgic-Radmanovic and L A Hetem and F Vasconcellos and J Maass and C Miranda and N Papaneophytou and J Raboch and A Fink-Jensen and A Okasha and J Korkeila and J D Guelfi and F Schneider and S Ohene and G Christodoulou and C R Soldatos and S K E Q Barrera and M Mendoza and R A Kallivayalil and S S Gudarzi and M R Lafta and M Bassi and M Clerici and R Gibson and T Kojima and S Nurmagambetova and S -C Cho and T Kadyrova and N Mikati and S Bajraktarov and T H Yen and B Ayushjav and L I Stevovic and J S S Molina and O Gureje and J O Johannessen and H R Chaudhry and B Al-Ashhab and A Araszkiewicz and D Prelipceanu and V Krasnov and A Bogdanov and M Jasovic-Gasic and L Vavrusova and P Pregelj and A F Liria and A Abdelrahman and P Udomratn and H Ulas and P Gokaip and F N Kigozi and G Richardson}, url = {https://www.scopus.com/inward/record.uri?eid=2-s2.0-79851492757&doi=10.1002%2fj.2051-5545.2010.tb00305.x&partnerID=40&md5=ebf47e1e84f22271aea10a73c93e9892}, doi = {10.1002/j.2051-5545.2010.tb00305.x}, issn = {17238617}, year = {2010}, date = {2010-01-01}, journal = {World Psychiatry}, volume = {9}, number = {3}, pages = {169-176}, publisher = {Blackwell Publishing Ltd}, abstract = {The treatment gap for people with mental disorders exceeds 50% in all countries of the world, approaching astonishingly high rates of 90% in the least resourced countries. We report the findings of the first systematic survey of leaders of psychiatry in nearly 60 countries on the strategies for reducing the treatment gap. We sought to elicit the views of these representatives on the roles of different human resources and health care settings in delivering care and on the importance of a range of strategies to increase the coverage of evidence-based treatments for priority mental disorders for each demographic stage (childhood, adolescence, adulthood and old age). Our findings clearly indicate three strategies for reducing the treatment gap: increasing the numbers of psychiatrists and other mental health professionals; increasing the involvement of a range of appropriately trained non-specialist providers; and the active involvement of people affected by mental disorders. This is true for both high income and low/middle income countries, though relatively of more importance in the latter. We view this survey as a critically important first step in ascertaining the position of psychiatrists, one of the most influential stakeholder communities in global mental health, in addressing the global challenge of scaling up mental health services to reduce the treatment gap.}, note = {cited By 127}, keywords = {Anxiety Disorder, Article, Atomoxetine, Atypical Antipsychotic Agent, Autism, Benzodiazepine, Bipolar Disorder, Central Nervous System Stimulants, Cholinesterase Inhibitor, Cognitive Therapy, Community Mental Health Center, Conduct Disorder, Cost Effectiveness Analysis, Dementia, Depression, Evidence-based Practice, Family, Family Therapy, Haloperidol, Health Care, Health Care Access, Health Care Delivery, Health Care Personnel, Health Practitioner, Health Survey, Help Seeking Behavior, Home Mental Health Care, Human, Hyperkinesia, Long Term Care, Lowest Income Group, Mental Deficiency, Mental Disease, Mental Health, Mental Health Care, Mental Health Service, Nootropic Agent, Open Ended Questionnaire, Outcome Assessment, Patient Compliance, Personality Disorder, Practice Guideline, Priority Journal, Psychiatry, Psychoeducation, Psychotherapy, Schizophrenia, Serotonin Noradrenalin Reuptake Inhibitor, Serotonin Uptake Inhibitor, Substance Abuse, Therapy, Therapy Delay, Tricyclic Antidepressant Agent, World Health Organization}, pubstate = {published}, tppubtype = {article} } The treatment gap for people with mental disorders exceeds 50% in all countries of the world, approaching astonishingly high rates of 90% in the least resourced countries. We report the findings of the first systematic survey of leaders of psychiatry in nearly 60 countries on the strategies for reducing the treatment gap. We sought to elicit the views of these representatives on the roles of different human resources and health care settings in delivering care and on the importance of a range of strategies to increase the coverage of evidence-based treatments for priority mental disorders for each demographic stage (childhood, adolescence, adulthood and old age). Our findings clearly indicate three strategies for reducing the treatment gap: increasing the numbers of psychiatrists and other mental health professionals; increasing the involvement of a range of appropriately trained non-specialist providers; and the active involvement of people affected by mental disorders. This is true for both high income and low/middle income countries, though relatively of more importance in the latter. We view this survey as a critically important first step in ascertaining the position of psychiatrists, one of the most influential stakeholder communities in global mental health, in addressing the global challenge of scaling up mental health services to reduce the treatment gap. |
Testingadminnaacuitm2020-05-28T06:49:14+00:00
2019 |
Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1 Journal Article Molecular Therapy - Methods and Clinical Development, 15 , pp. 18-26, 2019, ISSN: 23290501, (cited By 2). |
2010 |
Reducing the treatment gap for mental disorders: A WPA survey Journal Article World Psychiatry, 9 (3), pp. 169-176, 2010, ISSN: 17238617, (cited By 127). |