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Home > Past Conferences > Gene-based Therapeutics for Neurological Disorders

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Gene-based Therapeutics for Neurological Disorders

From Basic Science to Clinical Applications

08 Feb 2014 - 11 Feb 2014

Cancun, Mexico


This conference has now passed. Join us in Cancun in 2018 for the 2nd series, view details here. Alternatively, the following meeting may be of interest;

Neuroepigenetics and Neuroepitranscriptomics Conference | 24-27 Feb 2018 | Cancun, Mexico
Targeting Therapy of Alzheimer’s and Related Neurodegenerative Diseases | 01-04 June 2018 | Nassau Bahamas
3rd Interventions in Aging Conference | 03-06 Mar 2019 | Nassau Bahamas

This is a great opportunity to assemble a rich programme of international speakers to address the latest advances in gene therapy, from basic research of vector design, modelling and pre-clinical testing to clinical applications in CNS disorders such as Parkinson’s, Alzheimer’s, Huntington’s, motor neuron diseases and eye diseases.

The conference will bring together basic and clinical scientists to discuss recent discoveries in genetics and mechanisms of disease (e.g. the role of RNA metabolism and processing in neurological diseases, including amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), spinal muscular atrophy (SMA), and repeat expansion disorders) and how these discoveries could help develop novel gene based therapeutics for neurodegenerative diseases. Further topics such as challenges in vector manufacturing and views of regulatory agencies on gene therapy clinical applications will be discussed.

Confirmed Plenary Speakers

Professor Richard Jude Samulski
University of North Carolina at Chapel Hill

Professor Jeffrey H. Kordower
Rush University, Chicago

Professor Ronald G Crystal
Weill Cornell Medical College

Dr Krystof Bankiewicz University of California, San Francisco


Confirmed Invited Speakers

Dr Fraser Wright
Childrens Hospital of Philadelphia

Dr Maria Kinali
Imperial College, London

Bernard Schneider
Ecole Polytechnique Fédérale de Lausanne

Professor Alberto Auricchio

Dr. Hicham Fenniri

Prof. Giuseppe Battagli
University College London

Bas Blits
uniQure B.V.

Dr Howard Federoff
Georgetown University

Dr Daniel Lipinsky
University of Oxford

Joost Verhaagen
Netherlands Institute for Neuroscience

Dr Brian Kaspar
Nationwide Children's Hospital, Columbus












Saturday 8th February 2014

08:15 - 10:15

Registration & Reception


10:15 - 10:30

Opening  Comments


Session Title: Gene Therapy Approaches for Amyotrophic Lateral Sclerosis
Session Chair Ronald Crystal & Nicholas Mazarakis

10:30 – 11:15

Plenary: Ronald Crystal
Weill Cornell Medical College

Gene Therapy For CNS Disorders

11:15 – 11:50

Brian Kaspar
The Research Institute at Nationwide Children's Hospital

AAV9-mediated SOD1 downregulation as a Future Therapy for Amyotrophic Lateral Sclerosis

11:50 – 12:20

Bernard Schneider

Ecole Polytechnique Federale de Lausanne

SOD1 Silencing in Motoneurons or Glia Rescues Neuromuscular Function in ALS Mice

12:20 – 12:45

Maria Grazia Biferi

Centre de Myologie, Paris

Modeling of UBIQUILIN2-Related ALS Using AAV10 Vectors

12:45 – 13:00

Discussion and Q & A

13:00 – 14:15

Welcome Reception/Lunch

14:15 – 17:30

Free Time

Session Title:  GMP Manufacturing For Clinical Gene Therapy
Session Chair Jude Samulski

17:30 – 18:10

J Fraser Wright

Children's Hospital of Philadelphia

Manufacturing And Characterizing Recombinant AAV For Parkinson’s Disease Clinical Trials


18:10 – 18:40

Bas Blitz

The Use Of AAV Vectors Produced By A GMP-Compliant And Scalable Production Platform For The Treatment Of Neurodegenerative Diseases

18:40 – 19:10


19:10 – 20:30

Debate: GMP Manufacturing, Progress and Challenges

Moderator: Ronald Crystal

Panel: J Fraser Wright, Bas Blits, Jude Samulski

20:30 – 21:15

Cocktails and Networking

21:15 – 22:30

Dinner & Free Time

Sunday 9th February 2014

08:20 -08:30

Chair Opening

Session Title: Gene Therapy For Spinal Muscular Atrophy
Session Chairs: Brian Kaspar and Mimoun Azzouz

08:30 – 09:10

Maria Kinali

Imperial College London

Spinal Muscular Atrophy: a clinician's perspective on the diagnostic and management challenges


09:10 -09:40

Brian Kaspar
The Research Institute at Nationwide Children's Hospital

AAV9-Mediated systemic gene therapy for SMA


09:40 – 10:10

Martine Barkats
Centre de Myologie, Paris

Comparaison of Central and Peripheral scAAV9-SMN Gene Delivery for the Treatment of Spinal Muscular Atrophy

10:10 – 10:40

Ravindra Singh

Iowa State University

The Evolving Structural Map Of Therapeutic Targets For Splicing Correction In Spinal Muscular Atrophy

10:40 – 11:10


11:10 – 13:00

Round Table Debate: Clinical Gene Therapy for SMA: Hype or Hope?

Moderator: Jeffrey Kordower

13:00 – 18:00

Group Tour: Tulum (To be Confirmed)

19:00 – 22:00

Dinner & Free Time

Monday 10th February 2014

08:20 – 08:30

Chair Opening

Session Title: Parkinson’s Disease
Session Chair Krystof Bankiewicz

08:30 – 09:15

Jeffrey  Kordower
Rush University

Trophic Factor Gene Therapy for Parkinson’s Disease: Promises and Challenges

09:15 – 09:50

Krystof Bankiewicz

Trophic or Dopamine replacement Gene Transfer in PD; Pros and Cons

09:50 – 10:25

Howard Federoff
Georgetown University

In vitro and in vivo evaluation of a LRRK2G2019S allele-specific shRNA as potential gene therapy for human Parkinson’s disease

10:25 – 11:00


Session Title: Gene Therapy Vector Delivery & Emerging Non-Viral Technologies
Session Chair Alberto Auricchio & Mimoun Azzouz



11:00 – 11:45

Krystof Bankiewicz

Influence of Viral Vector Delivery on Outcome of CNS Gene Therapy Trials

11:45 – 12:20

Nicholas Boulis
Emory University

Delivery of Genes That Control Neural Activity in Functional Disorders

12:20 – 12:40

Denis Cecchin
UCL London

Polymer Nanaparticles Mediated Gene Delivery

12:40 – 16:15

Lunch & Free Time

16:15 – 17:00

Mark Tuszynski

University of California, San Diego

Discussion with Q&A –

Growth Factor Gene Therapy for Alzheimer’s Disease

17:00 – 17:30


17:30 – 18:15

Jude Samulski

University of North Carolina

AAV: Progress and Challenges

Session Title: Other Gene Therapy Applications for CNS Disorders
Session Chair Jeffrey Kordower & Jude Samulski

18:15 – 18:45

Olivier Danos


Intracerebral Administration of AAVrh.10 Carrying Human SGSH And SUMF1 cDNAS In Children With MPSIIIA Disease: Results Of A Phase I/II Trial

18:45 – 19:15

Joost Verhaagen

Netherlands Institute for Neuroscience

Gene Therapy To Promote Repair Of The Traumatically Injured Peripheral Nerve

19:15 – 19:45

Joseph C Glorioso

University of Pittsburgh

HSV Vectors and the Treatment of Chronic Pain

19:45 – 20:00

Discussion and Q&A


21:00 – 00:00

*Gala Night & Group Photo*

Tuesday 11th February 2014

09:30 - 9:45

Chair Opening

Session Title: New Gene Delivery Methods And Emerging Technologies
Session Chair Nicholas Boulis

09:45 – 10:30

Nicholas Mazarakis
Imperial College London

Lentiviral Vectors: Prospect for Clinical Gene Therapy

10:30 – 11:00

Luis Pereira de Almeida

University of Coimbra Portugal

Gene Silencing Strategy for MJD

11:00 – 11:20


11:20 – 11:55

Alberto Auricchio

TIGEM, Italy

Eye: An Attractive Target for Gene Therapeutics

11:55 – 12:30

Nicholas Boulis

Emory University School of Medicine

General Review

12:30 – 12:45

Closing Remarks (Chairs)

12:45 – 13:00

Farewell & Goodbye (Fusion)


Plenary Speakers

  • Dr. R. Jude Samulski
    Director, Gene Therapy Center, University of North Carolina at Chapel Hill
  • Dr Ronald Crystal
    Chairman, Department of Genetic Medicine, Weill Medical College of Cornell University
  • Professor Krystof Bankiewicz
    Professor, University of California San Francisco
  • Dr Jeffrey Kordower
    Professor, Rush University Medical Center

Invited Speakers

  • Dr Bas Blits
    Head neurobiology, uniQure
  • Dr. Bernard Schneider
    Research and Teaching Associate, Ecole Polytechnique Federale de Lausanne
  • Prof Joost Verhaagen
    Prof, Netherlands Institute for Neuroscience
  • Professor Alberto Auricchio
    Director of the Molecular Therapy (MT) Research Programme, TIGETelethon Institute of Genetics and Medicine (TIGEM)
  • Dr J Fraser Wright
    Associate Professor, Director Clinical Gene Therapy Core Facility, Children's Hospital of Philadelphia
  • Dr Maria Kinali
    Consultant paediatric Neurologist and Honorary Senior clinical Lecturer , Chelsea and Westminster Hospital
  • Dr. Howard Federoff
    Executive Vice President for Health Sciences and Executive Dean , Georgetown University Medical Center
  • Dr Olivier Danos
    Senior Vice President, Kadmon
  • Dr Martine Barkats
    "Biotherapy of Motor Neuron Disorders" Team Leader, Centre de Myologie, Inserm UMR974, Paris
  • Dr. Brian Kaspar
    Associate Professor, The Research Institute at Nationwide Children's Hospital
  • Dr. Nicholas Boulis
    Associate Professo rof Neurological Surgery, Emory University School of Medicine
  • Prof Luis Pereira de Almeida
    Prof., CNC - Center for Neuroscience and Cell Biology, University of Coimbra
  • Dr. Mark Tuszynski
    Professor, University of California, San Diego


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