David Segal, PhD Many people have heard of CRISPR as a tool to edit genes. This talk will discuss what CRISPR is and how it Share Your Feedback or Recommend New Topics ( ASF Dr. Allyson Berent, Angelman Syndrome Therapeutics | ASF Day of Learning 2025.
Advocating for Access: How Policy Shapes the Future of Gene Therapy MVX 220: A Gene Replacement Therapy for Angelman Syndrome Christopher Luthers (HSC-GT) | 2024 FAST Global Summit on Angelman Syndrome
Foundation for Angelman Syndrome Therapeutics presents "Collaborative efforts toward an AAV gene replacement therapy for Five gene therapy strategies are in development to cure Angelman syndrome. Visit your FAST friend's fundraising page or
2015 Global Summit on Angelman Syndrome: Friday panels - Kevin Nash, Ph.D. #10 FAST Advisory Council member, Dr. Terry Jo Bichell, interviews Dr. Ralf Schmid, PhD in the latest edition of Angelman Updates!
An Inside Look at Angelman Syndrome Rare Disease: An Interview with Amanda Moore Angelman syndrome symposium: Dr. Elizabeth Jalazo
Genetic Approaches for Treating Angelman Syndrome Gene Therapy for Angelman Syndrome Through RNA Interference Edwin Weeber, PhD. and Jodi A. Cook, PhD discuss utilization of the mouse and rat models for Angelman syndrome has allowed
The Use of GTX 102, an ASO as a Potential Therapeutic for Angelman Syndrome Dr Scott Stromatt 2020 Stormy Chamberlain, PhD, from the University of Connecticut discusses the recently announced research collaboration between
Our study shows that targeted genomic integration of a gene therapy vector can restore the function of paternally inherited UBE3A throughout life. In this episode: - How gene therapy works - The conditions for which gene therapy is currently approved - The future direction of
Jennifer Panagoulias, RAC | Transformatx Biotherapeutics Hematopoeitic Stem Cell Gene Therapy: What is ube-cel? For more Heartbreak to Hope: A Mother's Mission to Cure Angelman Syndrome | Dr Allyson Berent, CSO of FAST 2015 Global Summit on Angelman Syndrome: Friday panels - Agilis Biotherapeutics #3
Disclaimer: The information contained in these slides is provided for informational purposes only and should not be construed as Overview of the FAST research agenda for gene therapy and genetic editing in Angelman syndrome
Jennifer Panagoulias (Part 2) | 2022 FAST Summit on Angelman Syndrome MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today announced One avenue of treatment for AS is the use of gene therapy to reintroduce a functional copy of the UBE3A gene. Our group had previously shown
Timothy Yu, MD, PhD., Arthur Beaudet, MD, PhD., James Wilson, MD, PhD., Meghan Miller, PhD., Michelle Krishnan, MD, PhD., 2018 FAST Science Summit - Gene Therapy for Angelman Syndrome
In this episode, Amanda Moore, CEO of Angelman Syndrome Foundation speaks with Steve Smith, WCG's President of Patient 2019 FAST Summit on Angelman Syndrome - AAV Gene Therapy - Dr. Edwin Weeber, PTC Therapuetics Angelman Syndrome - Gene therapy
Dr. Kevin Nash explains gene therapy strategies for the treatment of Angelman Syndrome. This discussion will focus on AAV, A therapeutic strategy that addresses the root cause of this genetic disorder and holds great promise is the reactivation of the silenced
Background on PTC and their Gene Therapy Program in Angelman Syndrome Dr Stephanie Ciarlone 2020 F 2019 FAST Summit on Angelman Syndrome - HSC Therapy - Dr. Joe Anderson, Dr. Mehrdad Abedi, UC Davis
Stephanie Ciarlone | 2022 FAST Summit on Angelman Syndrome Angelman Syndrome IPSC and Brain Organoid Biorepository Dr Yong Hui Jiang 2020 FAST Science Summit
Jan Nolta, Ph.D., discusses the different platforms at the Stem Cell Program and Gene Therapy Center at the University of 2018 FAST Science Summit - Angelman Overview
Stormy Chamberlain, PhD, Assistant Director for University of Connecticut's Graduate Program in Genetics and Developmental 2018 FAST Science Summit - Round Table Panel on the Treatment of Angelman Syndrome
Hematopoietic Stem Cell Gene Therapy for Angelman Syndrome Be The Cure: ASF Funds Dr. Steven Gray Gene Therapy Research Keynote: AAV-mediated gene therapy to the central nervous system: prospect for Angelman syndrome
Allyson's Journey Joseph Anderson, Ph.D. and Mehrdad Abedi, M.D. The proof of concept work on utilizing a lentiviral vector hematopoietic stem Functional Rescue in an Angelman Syndrome Preclinical Model by Anna Adhikari
With James M. Wilson, M.D., Ph.D. Streamed live on December 1, 2017 from Chicago, IL. Disclaimer: The information contained in A kind of gene therapy, this potential treatment could lead to proper protein and cell function for individuals with Angelman syndrome. "This
The Foundation for Angelman Syndrome Therapeutics (FAST), according to Jennifer Panagoulias, is named that because they 2018 FAST Science Summit - Blood Stem Cells: New Gene Therapy Approach for Angelman Syndrome James M. Wilson | 2022 FAST Summit on Angelman Syndrome
Angelman Updates with Dr. Terry Jo Bichell, featuring Dr. Ralf Schmid Overview of Angelman syndrome presented by Allyson Berent, DVM, chief science officer, FAST.
Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients Genetics of Angelman syndrome Dr. Elizabeth Jalazo Director, Clinical Integration, Angelman Syndrome Foundation, Aurora,
Overview of the Therapeutic Landscape for Angelman Syndrome Dr. David Segal: Molecular Therapies in Angelman Syndrome (Friday afternoon panel)
UC Davis scientists pioneer stem cell gene therapy for Angelman Foundation for Angelman Syndrome Therapeutics presents "A therapeutic approach to treating Angelman syndrome using
Angelman Syndrome A Mother's Journey to a Cure. "There is currently no approved disease modifying treatment for Angelman syndrome, which results in profound impairment in individuals living
2019 FAST Summit on Angelman Syndrome - Genetics and Therapeutic Overview, Allyson Berent, CSO FAST Allyson Berent, DVM, DACVIM, Chief Science Officer for FAST, gives an overview of FAST's mission and vision through an
2018 FAST Science Summit - CRISPR - Pros and Cons, Promise, Possibilities, and Concerns Hematopoietic Stem Cell Gene Therapy for the Potential Treatment of Angelman Syndrome Christopher Luthers University of Allyson Berent provides an overview of the cause of Angelman syndrome and discusses the various potential therapeutic
A therapeutic approach where a harmless version of a virus, Adeno-Associated Virus (AAV), is used to carry a healthy copy of a gene to a target organ. This is Dr Theodora Markati on the Angelman Syndrome Therapies in Development
James M. Wilson, MD, PhD | Perelman School of Medicine, University of Pennsylvania hUBE3 A-AAV9 Gene Replacement Gene Therapy 101 with Dr. Kevin Nash For more information on spasticity or #YaleMedicine, visit:
In this part of the webinar, Dora Markati from Oxford University talks about various therapies in preclinical and clinical Ryan Butler, Ph.D. examines the potential for vectorized shRNA to induce paternal expression of UBE3A through the
In 2015 Dr. Berent became the Chief Science Officer for the Foundation for Angelman Syndrome Therapeutics and has been Stephanie Ciarlone, PhD | PTC Therapeutics PTC-AS Gene Therapy Program Update For more information, visit Watch this video for the latest Angelman syndrome news
Disclaimer: The information contained in these slides and video is provided for informational purposes only and should not be 2019 FAST Science Summit, AAV GeneTherapy, Dr. Jim Wilson, UPenn and John Crowley, Amicus
Friday's event schedule is as follows: 9:00 AM to 10:00 AM - New Animal Models in Angelman Syndrome - Featured Speakers: Dr. Full Title: Functional Rescue in an Angelman Syndrome Preclinical Model Following Treatment with Lentiviral Vector Transduced Researchers identify potential treatment for Angelman syndrome
Sharyl Fyffe-Maricich | Executive Director of Molecular and Cell Biology Research at Ultragenyx Pharmaceutical Inc. Gene Angelman Syndrome - Yale Medicine Explains Joe Anderson, PhD Stem cell gene therapy has the potential to treat Angelman syndrome. By genetically modifying a patient's
10 Deadly Sins|Angelman Syndrome FAST - Foundation for Angelman Syndrome Therapeutics Keynote address: Stem Cell & Gene Therapy Platforms
Dr. Edwin Weeber and Dr. Stephanie Ciarlone of PTC Therapeutics provide an overview of PTC and their GT-AS program. Gene Therapy Shows Early Promise as Angelman Syndrome Led by Ben Philpot, PhD, and Matt Judson, PhD, the new therapy was generally well-tolerated and prevented key signs of the condition in animal models.
Therapeutic Strategies for the Treatment of Angelman syndrome Dr Allyson Berent 2020 FAST Science Program Update on Hematopoietic Stem Cell Gene Therapy for Angelman Syndrome Dr Joe Anderson 2020 by James M Wilson, MD, PhD, GEMMABio and Elizabeth Berry-Kravis, MD, PhD, Rush Medical Center.
There is no effective treatment or cure. Angelman syndrome is caused by the loss of functional UBE3A gene in the brain. The gene provides The U.S. Food and Drug Administration (FDA) has given Mavrix Bio the go-ahead to start a first-in-human clinical trial testing MVX-220,
Ovid Therapeutics Collaborating with UConn to Develop Angelman Syndrome Treatment Potential shRNA Gene Therapy for Angelman Syndrome
Gene editing and gene therapies in genetically-defined autisms Come and join our latest series about "10 DEADLY SINS". Prepare to embark on a captivating journey into the enigmatic world of Improving Gene Therapy for Angelman Syndrome with Secreted
First-in-human study of Angelman gene therapy set for this year The Therapeutic Window For Angelman Syndrome Patients May Be With Allyson Berent, DVM, DACVIM, chief science office FAST Streamed live on December 1, 2017 from Chicago, IL. Disclaimer:
Jim Wilson, MD, PhD Updates on the gene replacement therapy program using AAV delivery for the treatment of Angelman Moving FAST to Treat Angelman Syndrome | Vital Science Podcast: S4, E08
Angelman Updates with Dr. Terry Jo Bichell, featuring Dr. Joe Anderson MavriX Bio Announces New Potential Gene Therapy for Angelman Jim Wilson, M.D., Ph.D., describes 3 different platforms his team is investigating to treat Angelman syndrome in a murine model.
FAST Advisory Council member, Dr. Terry Jo Bichell, interviews Dr. Joseph Anderson, Ph.D., Associate Professor at @UCDavis in Presenters: Mark J. Pykett, V.M.D, Ph.D., President and Chief Executive Officer of Agilis Biotherapeutics LLC Jodi Cook, Ph.D.,
Sharyl Fyffe Maricich | 2022 FAST Summit on Angelman Syndrome Presentations from the FAST Fire Team: the most aggressive, novel, innovative research initiative whose goal is to identify Cas9 gene therapy for Angelman syndrome traps Ube3a-ATS long
2018 FAST Science Summit - PTC Therapeutics - Gene Therapy for Angelman Syndrome