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Terapia genica con CERE-120 a 5 anni

provette ricercaRisultati positivi sulla sicurezza in 53 pazienti

CERE-120 è una terapia genica, che consiste nella iniezione nel putamen (un nucleo cerebrale affetto nel Parkinson) e nella sostanza nera tramite chirurgia stereotassica del gene per un fattore di crescita (Neurturina) tramite un virus reso innocuo. Si spera che il fattore di crescita possa arrestare o almeno rallentare il processo degenerativo.

Sono stati seguiti regolarmente per 5 anni con visite ogni 6 mesi (esame neurologico e clinico generale, esami di laboratorio di routine e acquisizione di immagini cerebrali tramite risonanza magnetica) 53 pazienti che avevano ricevuto la terapia e non sono stati segnalati problemi di sicurezza. La funzione motoria si è stabilizzata con risultati variabili da un lieve miglioramento del punteggio motorio UPDRS (3,2%) ad un peggioramento (-14,2%) rispetto a prima della terapia. I dati non sono utilizzabili, però, per la dimostrazione di efficacia, perché non sono stati raccolti in cieco rispetto ad un gruppo di controllo.

 

Fonte: Marks WJ e coll del gruppo di studio su CERE-120 Human Gene Therapy 2016 27 (7) 522-527

 

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  • SELECT DISTINCT a.id, a.title, a.name, a.checked_out, a.checked_out_time, a.note, a.state, a.access, a.created_time, a.created_user_id, a.ordering, a.language, a.fieldparams, a.params, a.type, a.default_value, a.context, a.group_id, a.label, a.description, a.required, a.only_use_in_subform,l.title AS language_title, l.image AS language_image,uc.name AS editor,ag.title AS access_level,ua.name AS author_name,g.title AS group_title, g.access as group_access, g.state AS group_state, g.note as group_note FROM jml_fields AS a LEFT JOIN `jml_languages` AS l ON l.lang_code = a.language LEFT JOIN jml_users AS uc ON uc.id=a.checked_out LEFT JOIN jml_viewlevels AS ag ON ag.id = a.access LEFT JOIN jml_users AS ua ON ua.id = a.created_user_id LEFT JOIN jml_fields_groups AS g ON g.id = a.group_id LEFT JOIN `jml_fields_categories` AS fc ON fc.field_id = a.id WHERE ( (`a`.`context` = :context AND (`fc`.`category_id` IS NULL OR `fc`.`category_id` IN (:preparedArray1,:preparedArray2,:preparedArray3)) AND `a`.`access` IN (:preparedArray4,:preparedArray5)) AND (`a`.`group_id` = 0 OR `g`.`access` IN (:preparedArray6,:preparedArray7)) AND `a`.`state` = :state) AND (`a`.`group_id` = 0 OR `g`.`state` = :gstate) AND `a`.`only_use_in_subform` = :only_use_in_subform ORDER BY a.ordering ASC585μs6.37KBParams/libraries/src/MVC/Model/BaseDatabaseModel.php:166Copy
  • SELECT `c`.`id`,`c`.`asset_id`,`c`.`access`,`c`.`alias`,`c`.`checked_out`,`c`.`checked_out_time`,`c`.`created_time`,`c`.`created_user_id`,`c`.`description`,`c`.`extension`,`c`.`hits`,`c`.`language`,`c`.`level`,`c`.`lft`,`c`.`metadata`,`c`.`metadesc`,`c`.`metakey`,`c`.`modified_time`,`c`.`note`,`c`.`params`,`c`.`parent_id`,`c`.`path`,`c`.`published`,`c`.`rgt`,`c`.`title`,`c`.`modified_user_id`,`c`.`version`, CASE WHEN CHAR_LENGTH(`c`.`alias`) != 0 THEN CONCAT_WS(':', `c`.`id`, `c`.`alias`) ELSE `c`.`id` END as `slug` FROM `jml_categories` AS `s` INNER JOIN `jml_categories` AS `c` ON (`s`.`lft` < `c`.`lft` AND `c`.`lft` < `s`.`rgt` AND `c`.`language` IN (:preparedArray3,:preparedArray4)) OR (`c`.`lft` <= `s`.`lft` AND `s`.`rgt` <= `c`.`rgt`) WHERE (`c`.`extension` = :extension OR `c`.`extension` = 'system') AND `c`.`access` IN (:preparedArray1,:preparedArray2) AND `c`.`published` = 1 AND `s`.`id` = :id ORDER BY `c`.`lft`586μs6.9KBParams/libraries/src/Categories/Categories.php:375Copy
  • SELECT custom_data FROM jml_extensions WHERE element = 'yootheme' AND folder = 'system' LIMIT 1336μs64.88KB/templates/yootheme/packages/platform-joomla/src/Storage.php:30Copy
  • SELECT `m`.`id`,`m`.`title`,`m`.`module`,`m`.`position`,`m`.`content`,`m`.`showtitle`,`m`.`params`,`mm`.`menuid` FROM `jml_modules` AS `m` LEFT JOIN `jml_modules_menu` AS `mm` ON `mm`.`moduleid` = `m`.`id` LEFT JOIN `jml_extensions` AS `e` ON `e`.`element` = `m`.`module` AND `e`.`client_id` = `m`.`client_id` WHERE ( ( (`m`.`published` = 1 AND `e`.`enabled` = 1 AND `m`.`client_id` = :clientId AND `m`.`access` IN (:preparedArray1,:preparedArray2)) AND (`m`.`publish_up` IS NULL OR `m`.`publish_up` <= :publishUp)) AND (`m`.`publish_down` IS NULL OR `m`.`publish_down` >= :publishDown)) AND (`mm`.`menuid` = :itemId OR `mm`.`menuid` <= 0) AND `m`.`language` IN (:preparedArray3,:preparedArray4) ORDER BY `m`.`position`,`m`.`ordering`2.7ms132.25KBParams/libraries/src/Cache/Controller/CallbackController.php:51Copy
  • SELECT `metakey` FROM `jml_content` WHERE `id` = :id153μs1.67KBParams/modules/mod_related_items/src/Helper/RelatedItemsHelper.php:88Copy
  • SELECT `a`.`id` FROM `jml_content` AS `a` WHERE ( ( (`a`.`id` != :id AND `a`.`state` = 1 AND `a`.`access` IN (:preparedArray1,:preparedArray2)) AND (`a`.`metakey` LIKE :preparedArray3 OR `a`.`metakey` LIKE :preparedArray4 OR `a`.`metakey` LIKE :preparedArray5 OR `a`.`metakey` LIKE :preparedArray6 OR `a`.`metakey` LIKE :preparedArray7)) AND (`a`.`publish_up` IS NULL OR `a`.`publish_up` <= :nowDate1)) AND (`a`.`publish_down` IS NULL OR `a`.`publish_down` >= :nowDate2) AND `a`.`language` IN (:preparedArray8,:preparedArray9) LIMIT 20494μs3.34KBParams/modules/mod_related_items/src/Helper/RelatedItemsHelper.php:152Copy
  • SELECT `a`.`id`,`a`.`title`,`a`.`alias`,`a`.`introtext`,`a`.`fulltext`,`a`.`checked_out`,`a`.`checked_out_time`,`a`.`catid`,`a`.`created`,`a`.`created_by`,`a`.`created_by_alias`,`a`.`modified`,`a`.`modified_by`,CASE WHEN `a`.`publish_up` IS NULL THEN `a`.`created` ELSE `a`.`publish_up` END AS `publish_up`,`a`.`publish_down`,`a`.`images`,`a`.`urls`,`a`.`attribs`,`a`.`metadata`,`a`.`metakey`,`a`.`metadesc`,`a`.`access`,`a`.`hits`,`a`.`featured`,`a`.`language`,LENGTH(`a`.`fulltext`) AS `readmore`,`a`.`ordering`,`fp`.`featured_up`,`fp`.`featured_down`,CASE WHEN `c`.`published` = 2 AND `a`.`state` > 0 THEN 2 WHEN `c`.`published` != 1 THEN 0 ELSE `a`.`state` END AS `state`,`c`.`title` AS `category_title`,`c`.`path` AS `category_route`,`c`.`access` AS `category_access`,`c`.`alias` AS `category_alias`,`c`.`language` AS `category_language`,`c`.`published`,`c`.`published` AS `parents_published`,`c`.`lft`,CASE WHEN `a`.`created_by_alias` > ' ' THEN `a`.`created_by_alias` ELSE `ua`.`name` END AS `author`,`ua`.`email` AS `author_email`,`uam`.`name` AS `modified_by_name`,`parent`.`title` AS `parent_title`,`parent`.`id` AS `parent_id`,`parent`.`path` AS `parent_route`,`parent`.`alias` AS `parent_alias`,`parent`.`language` AS `parent_language`,COALESCE(NULLIF(ROUND(`v`.`rating_sum` / `v`.`rating_count`, 1), 0), 0) AS `rating`,COALESCE(NULLIF(`v`.`rating_count`, 0), 0) AS `rating_count` FROM `jml_content` AS `a` LEFT JOIN `jml_categories` AS `c` ON `c`.`id` = `a`.`catid` LEFT JOIN `jml_users` AS `ua` ON `ua`.`id` = `a`.`created_by` LEFT JOIN `jml_users` AS `uam` ON `uam`.`id` = `a`.`modified_by` LEFT JOIN `jml_categories` AS `parent` ON `parent`.`id` = `c`.`parent_id` LEFT JOIN `jml_content_frontpage` AS `fp` ON `fp`.`content_id` = `a`.`id` LEFT JOIN `jml_content_rating` AS `v` ON `a`.`id` = `v`.`content_id` WHERE `a`.`access` IN (:preparedArray1,:preparedArray2) AND `c`.`access` IN (:preparedArray3,:preparedArray4) AND `c`.`published` = 1 AND `a`.`state` = :condition AND `a`.`id` IN (:preparedArray5,:preparedArray6,:preparedArray7,:preparedArray8,:preparedArray9,:preparedArray10,:preparedArray11,:preparedArray12,:preparedArray13,:preparedArray14,:preparedArray15,:preparedArray16,:preparedArray17,:preparedArray18,:preparedArray19,:preparedArray20,:preparedArray21,:preparedArray22,:preparedArray23,:preparedArray24) AND (`a`.`publish_up` IS NULL OR `a`.`publish_up` <= :publishUp) AND (`a`.`publish_down` IS NULL OR `a`.`publish_down` >= :publishDown) ORDER BY a.ordering ASC1.94ms332.03KBParams/libraries/src/MVC/Model/BaseDatabaseModel.php:166Copy
  • SELECT `c`.`id`,`c`.`asset_id`,`c`.`access`,`c`.`alias`,`c`.`checked_out`,`c`.`checked_out_time`,`c`.`created_time`,`c`.`created_user_id`,`c`.`description`,`c`.`extension`,`c`.`hits`,`c`.`language`,`c`.`level`,`c`.`lft`,`c`.`metadata`,`c`.`metadesc`,`c`.`metakey`,`c`.`modified_time`,`c`.`note`,`c`.`params`,`c`.`parent_id`,`c`.`path`,`c`.`published`,`c`.`rgt`,`c`.`title`,`c`.`modified_user_id`,`c`.`version`, CASE WHEN CHAR_LENGTH(`c`.`alias`) != 0 THEN CONCAT_WS(':', `c`.`id`, `c`.`alias`) ELSE `c`.`id` END as `slug` FROM `jml_categories` AS `s` INNER JOIN `jml_categories` AS `c` ON (`s`.`lft` < `c`.`lft` AND `c`.`lft` < `s`.`rgt` AND `c`.`language` IN (:preparedArray3,:preparedArray4)) OR (`c`.`lft` <= `s`.`lft` AND `s`.`rgt` <= `c`.`rgt`) WHERE (`c`.`extension` = :extension OR `c`.`extension` = 'system') AND `c`.`access` IN (:preparedArray1,:preparedArray2) AND `c`.`published` = 1 AND `s`.`id` = :id ORDER BY `c`.`lft`409μs6.9KBParams/libraries/src/Categories/Categories.php:375Copy
  • SELECT `c`.`id`,`c`.`asset_id`,`c`.`access`,`c`.`alias`,`c`.`checked_out`,`c`.`checked_out_time`,`c`.`created_time`,`c`.`created_user_id`,`c`.`description`,`c`.`extension`,`c`.`hits`,`c`.`language`,`c`.`level`,`c`.`lft`,`c`.`metadata`,`c`.`metadesc`,`c`.`metakey`,`c`.`modified_time`,`c`.`note`,`c`.`params`,`c`.`parent_id`,`c`.`path`,`c`.`published`,`c`.`rgt`,`c`.`title`,`c`.`modified_user_id`,`c`.`version`, CASE WHEN CHAR_LENGTH(`c`.`alias`) != 0 THEN CONCAT_WS(':', `c`.`id`, `c`.`alias`) ELSE `c`.`id` END as `slug` FROM `jml_categories` AS `s` INNER JOIN `jml_categories` AS `c` ON (`s`.`lft` < `c`.`lft` AND `c`.`lft` < `s`.`rgt` AND `c`.`language` IN (:preparedArray3,:preparedArray4)) OR (`c`.`lft` <= `s`.`lft` AND `s`.`rgt` <= `c`.`rgt`) WHERE (`c`.`extension` = :extension OR `c`.`extension` = 'system') AND `c`.`access` IN (:preparedArray1,:preparedArray2) AND `c`.`published` = 1 AND `s`.`id` = :id ORDER BY `c`.`lft`347μs6.9KBParams/libraries/src/Categories/Categories.php:375Copy
  • SELECT `c`.`id`,`c`.`asset_id`,`c`.`access`,`c`.`alias`,`c`.`checked_out`,`c`.`checked_out_time`,`c`.`created_time`,`c`.`created_user_id`,`c`.`description`,`c`.`extension`,`c`.`hits`,`c`.`language`,`c`.`level`,`c`.`lft`,`c`.`metadata`,`c`.`metadesc`,`c`.`metakey`,`c`.`modified_time`,`c`.`note`,`c`.`params`,`c`.`parent_id`,`c`.`path`,`c`.`published`,`c`.`rgt`,`c`.`title`,`c`.`modified_user_id`,`c`.`version`, CASE WHEN CHAR_LENGTH(`c`.`alias`) != 0 THEN CONCAT_WS(':', `c`.`id`, `c`.`alias`) ELSE `c`.`id` END as `slug` FROM `jml_categories` AS `s` INNER JOIN `jml_categories` AS `c` ON (`s`.`lft` < `c`.`lft` AND `c`.`lft` < `s`.`rgt` AND `c`.`language` IN (:preparedArray3,:preparedArray4)) OR (`c`.`lft` <= `s`.`lft` AND `s`.`rgt` <= `c`.`rgt`) WHERE (`c`.`extension` = :extension OR `c`.`extension` = 'system') AND `c`.`access` IN (:preparedArray1,:preparedArray2) AND `c`.`published` = 1 AND `s`.`id` = :id ORDER BY `c`.`lft`334μs17.9KBParams/libraries/src/Categories/Categories.php:375Copy
  • SELECT `c`.`id`,`c`.`asset_id`,`c`.`access`,`c`.`alias`,`c`.`checked_out`,`c`.`checked_out_time`,`c`.`created_time`,`c`.`created_user_id`,`c`.`description`,`c`.`extension`,`c`.`hits`,`c`.`language`,`c`.`level`,`c`.`lft`,`c`.`metadata`,`c`.`metadesc`,`c`.`metakey`,`c`.`modified_time`,`c`.`note`,`c`.`params`,`c`.`parent_id`,`c`.`path`,`c`.`published`,`c`.`rgt`,`c`.`title`,`c`.`modified_user_id`,`c`.`version`, CASE WHEN CHAR_LENGTH(`c`.`alias`) != 0 THEN CONCAT_WS(':', `c`.`id`, `c`.`alias`) ELSE `c`.`id` END as `slug` FROM `jml_categories` AS `s` INNER JOIN `jml_categories` AS `c` ON (`s`.`lft` < `c`.`lft` AND `c`.`lft` < `s`.`rgt` AND `c`.`language` IN (:preparedArray3,:preparedArray4)) OR (`c`.`lft` <= `s`.`lft` AND `s`.`rgt` <= `c`.`rgt`) WHERE (`c`.`extension` = :extension OR `c`.`extension` = 'system') AND `c`.`access` IN (:preparedArray1,:preparedArray2) AND `c`.`published` = 1 AND `s`.`id` = :id ORDER BY `c`.`lft`321μs6.38KBParams/libraries/src/Categories/Categories.php:375Copy
  • SELECT `c`.`id`,`c`.`asset_id`,`c`.`access`,`c`.`alias`,`c`.`checked_out`,`c`.`checked_out_time`,`c`.`created_time`,`c`.`created_user_id`,`c`.`description`,`c`.`extension`,`c`.`hits`,`c`.`language`,`c`.`level`,`c`.`lft`,`c`.`metadata`,`c`.`metadesc`,`c`.`metakey`,`c`.`modified_time`,`c`.`note`,`c`.`params`,`c`.`parent_id`,`c`.`path`,`c`.`published`,`c`.`rgt`,`c`.`title`,`c`.`modified_user_id`,`c`.`version`, CASE WHEN CHAR_LENGTH(`c`.`alias`) != 0 THEN CONCAT_WS(':', `c`.`id`, `c`.`alias`) ELSE `c`.`id` END as `slug` FROM `jml_categories` AS `s` INNER JOIN `jml_categories` AS `c` ON (`s`.`lft` < `c`.`lft` AND `c`.`lft` < `s`.`rgt` AND `c`.`language` IN (:preparedArray3,:preparedArray4)) OR (`c`.`lft` <= `s`.`lft` AND `s`.`rgt` <= `c`.`rgt`) WHERE (`c`.`extension` = :extension OR `c`.`extension` = 'system') AND `c`.`access` IN (:preparedArray1,:preparedArray2) AND `c`.`published` = 1 AND `s`.`id` = :id ORDER BY `c`.`lft`324μs6.9KBParams/libraries/src/Categories/Categories.php:375Copy
  • SELECT `params` FROM `jml_comprofiler_plugin` WHERE `id` = 1170μs896BCopy
  • SELECT `id` FROM `jml_rstbox` WHERE `published` = 1 AND `testmode` = 0181μs928B/administrator/components/com_rstbox/EngageBox/Boxes.php:45Copy
  • SELECT * FROM `jml_rstbox` WHERE `id` = 3128μs2.23KB/administrator/components/com_rstbox/EngageBox/Box.php:238Copy
  • SELECT COUNT(id) FROM `jml_rstbox_logs` WHERE `event` = 1 AND `box` = 3 AND `visitorid` = '668f71bec38cd170' AND MONTH(date) = 03 AND YEAR(date) = 2025177μs896B/administrator/components/com_rstbox/EngageBox/Assignments.php:201Copy
  • SELECT * FROM `jml_rstbox` WHERE `id` = 5135μs2.23KB/administrator/components/com_rstbox/EngageBox/Box.php:238Copy
  • SELECT COUNT(id) FROM `jml_rstbox_logs` WHERE `event` = 1 AND `box` = 5 AND `visitorid` = '668f71bec38cd170'131μs896B/administrator/components/com_rstbox/EngageBox/Assignments.php:201Copy
  • SELECT `language`,`id` FROM `jml_menu` WHERE `home` = '1' AND `published` = 1 AND `client_id` = 0474μs976B/libraries/src/Language/Multilanguage.php:123Copy
  • SELECT `alias` FROM `jml_content` WHERE `id` = :id124μs1.67KBParams/components/com_content/src/Service/Router.php:168Copy
  • SELECT `alias` FROM `jml_content` WHERE `id` = :id87μs1.67KBParams/components/com_content/src/Service/Router.php:168Copy
  • SELECT `c2`.`language`,CONCAT_WS(':', `c2`.`id`, `c2`.`alias`) AS `id`,CONCAT_WS(':', `ca`.`id`, `ca`.`alias`) AS `catid` FROM `jml_content` AS `c` INNER JOIN `jml_associations` AS `a` ON `a`.`id` = `c`.`id` AND `a`.`context` = :context INNER JOIN `jml_associations` AS `a2` ON `a`.`key` = `a2`.`key` INNER JOIN `jml_content` AS `c2` ON `a2`.`id` = `c2`.`id` INNER JOIN `jml_categories` AS `ca` ON `c2`.`catid` = `ca`.`id` AND `ca`.`extension` = :extension2 WHERE `c`.`id` = :id AND c2.access IN (1,5) AND c2.language != 'it-IT' AND (c2.publish_up IS NULL OR c2.publish_up <= '2025-03-25 05:56:20') AND (c2.publish_down IS NULL OR c2.publish_down >= '2025-03-25 05:56:20') AND c2.state = 1313μs1.81KBParams/libraries/src/Language/Associations.php:145Copy
  • SELECT DISTINCT a.id, a.title, a.name, a.checked_out, a.checked_out_time, a.note, a.state, a.access, a.created_time, a.created_user_id, a.ordering, a.language, a.fieldparams, a.params, a.type, a.default_value, a.context, a.group_id, a.label, a.description, a.required, a.only_use_in_subform,l.title AS language_title, l.image AS language_image,uc.name AS editor,ag.title AS access_level,ua.name AS author_name,g.title AS group_title, g.access as group_access, g.state AS group_state, g.note as group_note FROM jml_fields AS a LEFT JOIN `jml_languages` AS l ON l.lang_code = a.language LEFT JOIN jml_users AS uc ON uc.id=a.checked_out LEFT JOIN jml_viewlevels AS ag ON ag.id = a.access LEFT JOIN jml_users AS ua ON ua.id = a.created_user_id LEFT JOIN jml_fields_groups AS g ON g.id = a.group_id WHERE ( (`a`.`context` = :context AND `a`.`access` IN (:preparedArray1,:preparedArray2)) AND (`a`.`group_id` = 0 OR `g`.`access` IN (:preparedArray3,:preparedArray4)) AND `a`.`state` = :state) AND (`a`.`group_id` = 0 OR `g`.`state` = :gstate) AND `a`.`only_use_in_subform` = :only_use_in_subform ORDER BY a.ordering ASC674μs5.63KBParams/libraries/src/MVC/Model/BaseDatabaseModel.php:166Copy
  • SELECT SUM(CASE WHEN `a`.`next_execution` <= :now THEN 1 ELSE 0 END) AS due_count,SUM(CASE WHEN `a`.`locked` IS NULL THEN 0 ELSE 1 END) AS locked_count FROM `jml_scheduler_tasks` AS `a` WHERE `a`.`state` = 1191μs1.75KBParams/administrator/components/com_scheduler/src/Model/TasksModel.php:517Copy
  • SHOW TABLES731μs8.83KB/libraries/vendor/joomla/database/src/Mysqli/MysqliDriver.php:678Copy
  • SELECT `session_id` FROM `jml_session` WHERE `session_id` = ?155μs1.67KBParams/libraries/vendor/joomla/session/src/Handler/DatabaseHandler.php:291Copy
  • UPDATE `jml_session` SET `data` = ? , `time` = ? WHERE `session_id` = ?8.81ms936BParams/libraries/vendor/joomla/session/src/Handler/DatabaseHandler.php:318Copy