1 00:00:08,919 --> 00:00:11,839 The Laboratory of Cell Regeneration and Plasticity 2 00:00:11,839 --> 00:00:15,320 is one of the PIGMOD Centre’s three laboratories. 3 00:00:15,640 --> 00:00:18,760 Our main objective is the biomedical research 4 00:00:18,760 --> 00:00:24,519 and preclinical testing of various therapeutic approaches in miniature pigs. 5 00:00:25,120 --> 00:00:27,800 The advantages of miniature pigs lie in their physiological and anatomical similarity 6 00:00:27,800 --> 00:00:33,079 to humans and the minimal ethical hurdles associated with their use. 7 00:00:33,560 --> 00:00:37,920 The minipigs represent a unique model for testing the safety 8 00:00:37,920 --> 00:00:42,000 and efficacy of treatment prior to clinical trials. 9 00:00:42,000 --> 00:00:46,520 We specialise in studying Huntington’s disease, spinal injuries, 10 00:00:46,520 --> 00:00:51,439 and eye disorders, i.e., retinopathies such as Usher syndrome, 11 00:00:51,439 --> 00:00:55,359 Stargardt disease and macular degeneration. 12 00:00:55,439 --> 00:01:01,520 "For Huntington’s disease study, we use two minipig models, transgenic and knock-in." 13 00:01:02,079 --> 00:01:05,799 Currently, we are working with the Technical University of Munich 14 00:01:05,799 --> 00:01:08,920 to create a humanised minipig model. 15 00:01:09,239 --> 00:01:16,000 By using behavioural, biochemical and molecular genetic methods on these models, 16 00:01:16,000 --> 00:01:19,280 we are trying to characterise the onset and course of the disease. 17 00:01:19,480 --> 00:01:22,120 In addition, we also use them for testing novel therapies. 18 00:01:22,760 --> 00:01:24,480 Our long-standing collaboration 19 00:01:24,480 --> 00:01:27,560 with various biotechnology and pharmaceutical companies 20 00:01:27,560 --> 00:01:32,560 involves preclinical studies aimed at reducing huntingtin levels. 21 00:01:33,000 --> 00:01:34,920 Based on our joint results, 22 00:01:34,920 --> 00:01:40,159 uniQure has been approved to conduct gene therapy clinical trials on humans. 23 00:01:40,359 --> 00:01:43,840 Additionally, we focus on the use of nanoparticles in gene therapy 24 00:01:43,840 --> 00:01:48,319 and look into the possibilities of regenerative medicine 25 00:01:48,319 --> 00:01:51,319 featuring neuronal reprogramming. 26 00:01:51,319 --> 00:01:56,239 „Currently, the PIGMOD Centre is developing a new gene therapy programme 27 00:01:56,239 --> 00:02:00,640 for severe eye disorders that remain untreatable.“ 28 00:02:00,640 --> 00:02:04,640 It concerns two serious diseases: 29 00:02:04,640 --> 00:02:10,039 Usher syndrome, which starts with a condition known as “tunnel vision”, 30 00:02:10,520 --> 00:02:13,919 We run this programme in collaboration 31 00:02:13,919 --> 00:02:18,319 with the German universities in Munich, Mainz and Giessen. 32 00:02:18,639 --> 00:02:22,199 "Age-related macular degeneration (AMD) is a neurodegenerative retinal disease 33 00:02:22,199 --> 00:02:25,159 and the leading cause of severe vision loss in people over the age of 60 34 00:02:25,159 --> 00:02:29,039 and affects hundreds of thousands of people in the Czech Republic." 35 00:02:29,319 --> 00:02:33,479 The dry form of this disease is still untreatable to this day. 36 00:02:33,479 --> 00:02:36,520 We are exploring cell therapy options 37 00:02:36,520 --> 00:02:40,680 involving the transplantation of healthy retinal cells into the affected eye. 38 00:02:40,680 --> 00:02:44,719 We insert the cultured cells into the eye on a nanofiber carrier, 39 00:02:44,719 --> 00:02:47,840 and, thanks to the similarity of the pig eye to the human eye, 40 00:02:47,840 --> 00:02:51,360 we can improve the technical performance of the transplantation 41 00:02:51,360 --> 00:02:53,199 and monitor its efficacy. 42 00:02:54,879 --> 00:02:58,439 Our research is helping to move therapies for incurable diseases 43 00:02:58,439 --> 00:03:01,400 from preclinical testing to the clinical stage. 44 00:03:01,400 --> 00:03:03,319 Thanks to the genetic pig models, 45 00:03:03,319 --> 00:03:06,520 we are at least able to mimic the damage 46 00:03:06,520 --> 00:03:10,000 and provide a platform for methods and testing of novel therapies. 47 00:03:10,000 --> 00:03:13,639 At the same time, we are actively involved in developing novel therapies. 48 00:03:13,639 --> 00:03:17,639 Our research is closely linked to patient organisations from around world. 49 00:03:17,639 --> 00:03:23,439 We travel to give lectures and receive funding from abroad. 50 00:03:23,439 --> 00:03:26,840 The trust patients put into our work is our greatest driving force.