Grants
CDKL5 Pilot Grant Programme Awardees 2022:
Joseph Anderson, PhD
University of California, Davis, Sacramento, CA USA
“Hematopoietic stem cell gene therapy as a therapeutic approach for CDKL5 Deficiency Disorder”
Davide Cacciarelli, PhD
Telethon Institute of Genetics and Medicine, Pozzuloli, ITALY
“A functional genomics approach to dissect the molecular bases of CDKL5 Deficiency Disorder”
M. Leonor Cancela, PhD
University of Algarve, Faro, PORTUGAL
“Rescuing functional defects in a zebrafish model of CDKL5 deficiency: Contribution to the identification of therapeutic target”
Diego Di Bernardo, PhD
Telethon Institute of Genetics and Medicine, Pozzuloli, ITALY
“Computational drug repurposing for CDKL5 Deficiency Disorder”
Mustafa Sahin, MD PhD
Boston Children’s Hospital, Boston, MA USA
“Utilizing patient iPSC-derived neurons to determine the functional consequences of longer cilia in CDKL5 Deficiency Disorder (CDD)”
CDKL5 Pilot Grant Programme Awardees 2021:
Michela Fagiolini, PhD
Boston Children's Hospital, Boston, MA USA
“ASO therapy in CDKL5 deficiency disorder”
Victor Faundez, PhD
Emory University, Atlanta, GA USA
“Systems Analysis of the CDKL5-Deficiency Disorder (CDKL5-DD) Cerebrospinal Fluid Proteome”
Michael J. Higley, MD, PhD
Yale University, New Haven, CT USA
“Impact of Cdkl5 deficiency on cortical dynamics and network architecture”
Peter K. Jackson, PhD
Stanford University School of Medicine, Stanford, CA USA
“Defining ciliary signaling and targets in CDKL5 Deficiency Disorder”
Christopher McGraw, MD, PhD
Boston Children's Hospital, Boston, MA USA
“Advancing a mosaic CDKL5 zebrafish model for high-throughput screening approaches”
Dolan Sondhi, PhD
Weill Cornell Medicine, New York, NY USA
“Comparison of Brain Biodistribution of Two CDKL5 Gene Therapy Vector Leads”
CDKL5 Pilot Grant Programme Awardees 2020:
Alan Brown, PhD
Harvard Medical School
“Visualizing the effect of CDKL5 deficiency on ciliary core structures”
Liqin Cao, PhD
Tsukuba University
“Elucidate the neural mechanisms underlying sleep disturbances and the relation between seizures and sleep-wake cycle in CDKL5 deficiency disorder (CDD)”
Simone DiGiovanni, MD, PhD
Imperial College London
“Characterization and correction of Cdkl5 nuclear function in human CDD neurons during maturation”
Maolin Guo, PhD
University of Massachusetts, Dartmouth
“Incorporation of functional unnatural amino acids into CDKL5 to study its function in cells”
Steven A. McCarroll, PhD
Harvard Medical School
“Ascertaining the cell-autonomous effects of CDKL5 mutations in every brain cell type”
Lauren Orefice, PhD
Massachusetts General Hospital
“Assessing peripheral sensory neuron dysfunction in animal models for CDKL5 deficiency disorder”
Gregory J. Pazour, PhD
University of Massachusetts Medical School
“CDKL5 regulation of ciliary assembly and signaling”
Maria Luisa Tutino, PhD
University of Naples
“Structural/functional characterization of full-length hCDKL5 isoform 1 produced in recombinant marine bacteria and use of pharmacological chaperones to stabilize hCDKL5 missense mutants”
CDKL5 Pilot Grant Programme Awardees 2019:
Massimiliano Bianchi, PhD
Trinity University, Dublin, Ireland
“Plasma microtubule proteins as potential biomarkers for CDKL5 Deficiency Disorder (CDD)”
Victor Faundez, PhD
Emory University
“CDKL5-Deficiency Disorder (CDD) Biomarkers Discovery Through System Biology”
Teruyuki Tanaka, PhD
University of Tokyo
“Identifying the brain-wide, synapse-to-circuit functional abnormalities upon the loss of CDKL5 kinase activity in ubiquitous/excitatory/inhibitory-neuron-specific Cdkl5 kinase-dead knock-in mice and human patients”
Cleber A. Trujillo, PhD
University of California, San Diego
“High-throughput drug screening platform for CDD cortical organoids”
Continuation Funding Awardees:
Alysson Muotri, PhD
University of California, San Diego
"CDKL5 Syndrome cortical organoids for drug testing and reversibility potential" (2017)
David Liu, PhD
“Development of Targeted Frameshifting Technologies” (2018)
CDKL5 Pilot Grant Programme Awardees 2018:
Nicola Allen, PhD
Salk Institute for Biological Sciences
"Systems Level Analysis of CDKL5 Astrocytes to Identify Novel Markers and Pathways in CDKL5 Deficiency Disorder (CDD)"
Alessia DiNardo, PhD
Boston Children’s Hospital
"High Content Screen to identify drug targets for the neuronal manifestations of CDKL5 deficiency"
James Eubanks, PhD
University of Toronto
"Evaluating a Novel Strategy to Stimulate mTORC1 in Two Mouse Models of CDKL5 Deficiency Disorder"
Michela Fagiolini, PhD
Boston Children’s Hospital
"Testing functional and structural connectivity in CDKL5 deficiency disorder as novel biomarkers"
Michael Greenberg, PhD
Harvard Medical School
"Single-cell Transcriptional Profiling of Mosaic CDKL5 Deficiency Disorder Brain Tissue"
David Liu, PhD
Broad Institute and Harvard University
"Development of Targeted Frameshifting Technologies"
Ihn Sik Seong, PhD
Massachusetts General Hospital
"Quantitative SRM assays for optimization of CDKL5 protein replacement therapy"
CDKL5 Pilot Grant Programme Awardees 2017:
Timothy Benke, MD, PhD
University of Colorado
"Mechanisms and treatment of paradoxical hyperexcitability in CDKL5 Deficiency Syndrome"
Elisabetta Ciani, PhD
University of Bologna
"Innovative Strategy to Enhance the Efficiency of Gene Therapy for CDKL5 Disorder"
Vera Kalscheuer, PhD
Max Planck Institute for Molecular Genetics
"Novel CDKL5 complex partners and kinase substrate candidates"
Charlotte Kilstrup-Nielsen, PhD
University of Insurbia
"Therapeutic potential of pregnenolone and its synthetic non-metabolized derivative for CDKL5 disorder"
Alysson Muotri, PhD
University of California San Diego
"CDKL5 Syndrome cortical organoids for drug testing and reversibility potential"
Tommaso Pizzorusso, PhD
Institute of Neuroscience, CNR
"Rescuing CDKL5 mice phenotype by targeting developmental critical period mechanisms"
David Rowitch, MD, PhD
University of Cambridge
"Understanding CDKL5 Expression Pattern by RNAScope in Developing Mouse and Human Glia"
W Andy Tao, PhD
Purdue University
"Identification of CDKL5 direct substrates based on kinase assay linked phosphoproteomics"
Sila Ultanir, PhD
The Francis Crick Institute
"Development of biomarkers for CDKL5 activity"
Robert Wilson, MD, PhD
University of Pennsylvania and the Children's Hospital of Philadelphia
"Therapeutic drug discovery for CDKL5 deficiency using random shRNA selection"
CDKL5 Program of Excellence 2016 Pilot Grant Awardees:
Bianca De Filippis, PhD
Instituto Superiore di Sanità
“Brain Rho GTPases: in search of innovative therapeutic targets for CDKL5 deficiency”
Michael Green, MD, PhD
University of Massachusetts Medical School
“Pharmacological Reactivation of the Xi-Linked CDKL5 Gene as a Potential Treatment for CDKL5 Deficiency”
Frances Jensen, MD, FACP
University of Pennsylvania
“Targeting the chloride-importing and exporting transporters in CDKL5 disorder”
Robin Kleiman, PhD
Boston Children’s Hospital
Michael Johnson, MD, PhD
Imperial College London
“Development of a High-content drug repurposing screen using CDKL5 patient derived iPSC neurons”
Charlotte Kilstrup-Nielsen, PhD
University of Insubria
“Predictive Drug Repurposing for CDKL5 Syndrome”
Nicoletta Landsberger, PhD
University of Milan
“Exploiting computational biology for target identification and drug repositioning in CDKL5 disorder”
Jeannie Lee, MD, PhD
Massachusetts General Hospital
“Treating CDKL5 Syndrome by reactivating the silent allele of CDKL5”
Jan Nolta, PhD
University of California, Davis Medical Center
“Development of TALE and CRISPR/Cas9 as a putative therapy for treatment or correction of genetically linked CDKL5 Deficiency”
Roy Perlis, MD, MSC
Massachusetts General Hospital
"Precision Medicine Program for CDKL5 Disorders: Therapeutic Screening Using Human iPSC Models”
Jianrong Tang, PhD
Baylor College of Medicine
“Studies of forniceal deep brain stimulation and hippocampal memory in CDKL5 mice”
Zhaolan Zhou, PhD
University of Pennsylvania
“Identifying a Neural Circuit Readout for CDKL5 Disorder”
Grants awarded in 2015:
Prof. John Rouse
MRC Protein Phosphorylation Unit
University of Dundee
“Identification of substrates, modes of regulation and cellular roles of the CDKL5 kinase” (2016-2018)
Prof. Peter Kind
Patrick Wild Centre
University of Edinburgh
“Understanding and rescuing core deficits underlying CDKL5” (2016-2018)
Including:
Disorder related phenotyping of CDKL5 knock out mice & rats and
Molecular and cellular phenotyping and rescue
Dr. Zhaolan (Joe) Zhou
University of Pennsylvania
“Genetic Dissection for CDKL5 Function in Mice” (2016-2017)
(Co-funded by Loulou Foundation 78.3% and IFCR 21.7%)
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Awards
2020
- CDKL5 Forum Award for Excellence - Lab of the Year 2020 – Samaco Lab, Baylor College of Medicine
- CDKL5 Forum Award for Excellence - Company Making a Difference 2020 Preclinical – Ultragenyx Pharmaceutical
- CDKL5 Forum Award for Excellence – Company Making a Difference 2020 Clinical – Marinus Pharmaceuticals
- CDKL5 Forum Award for Excellence – Champions of Progress 2020 – Helen Leonard and Jenny Downs, Telethon Kids Institute, Perth, Australia
CDKL5 Forum Junior Fellowship Award
- Theresa Bartels; Rowitch laboratory, University of Cambridge
- Regina Faubel, PhD; Lo laboratory, University of Pittsburgh
- Oliver Hardt, PhD; McGill University
- Paolo La Montanara, MD; Hospital ASL-Bari
- Ralf Schmid, PhD; Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania
2019
- CDKL5 Forum Award for Excellence - Lab of the Year 2019 – Muotri Lab, University of California, San Diego
- CDKL5 Forum Award for Excellence - Company Making a Difference 2019 Preclinical – Ultragenyx Pharmaceutical
- CDKL5 Forum Award for Excellence – Company Making a Difference 2019 Clinical – Zogenix
- CDKL5 Forum Award for Excellence – Champion of Progress 2019 – Timothy Benke, MD PhD, University of Colorado – Denver; Children’s Hospital Colorado
CDKL5 Forum Junior Fellowship Award
- Scott Demarest, MD; Pediatrics-Neurology, University of Colorado, Denver
- Julian Halmai, PhD; Fink laboratory, University of California, Davis
- James Nelson, PhD; Liu laboratory, Broad Institute
- Barbara Terzic; Zhou laboratory, University of Pennsylvania
- Nicole Van Bergen; Christodoulou laboratory, Murdoch Children’s Research Institute, Victoria, Australia
2018
- CDKL5 Forum Award for Excellence – Lab of the Year 2018 – Ultanir Lab, Francis Crick Institute
- CDKL5 Forum Award for Excellence – Company Making a Difference 2017 Preclinical – Takeda
- CDKL5 Forum Award for Excellence – Company Making a Difference 2017 Clinical – Ovid Therapeutics
- CDKL5 Forum Award for Excellence – Champion of Progress – the CDKL5 Alliance
CDKL5 Forum Junior Fellowships Award
- Isabella Barbiero – University of Insubria
- Paul Baxter – University of Edinburgh
- Priscilla Negraes – University of California, San Diego
- William Renthal – Harvard Medical School
- Madhumita Yennawar – University of Pennsylvania
2017
- CDKL5 Forum Award for Excellence – Lab of the Year 2017 – Zhou Lab
- CDKL5 Forum Award for Excellence – Company Making a Difference 2017 Preclinical – Amicus Therapeutics
- CDKL5 Forum Award for Excellence – Company Making a Difference 2017 Clinical – Marinus
- CDKL5 Forum Award for Excellence – Champion of Progress – the Howard Family
CDKL5 Forum Junior Fellowships Award
- Lucas Baltussen – Francis Crick Institute
- Claudia Fuchs – University of Bologna
- Ralph Hector – Glasgow University
- Ivan Munoz – University of Dundee
- Laura Rusconi – University of Insubria
2016
- CDKL5 Forum Award for Excellence – Lab of the Year 2016 – Devinsky Clinic, NYU
- CDKL5 Forum Award for Excellence – Company Making a Difference 2016 – PTC Therapeutics
- CDKL5 Forum Award for Excellence – Outstanding Contribution - Michael Jasulavic
2015
CDKL5 Forum Award for Excellence – Lab of the Year 2015 – Ciani Lab, University of Bologna
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