ALS Research Projects: Grants on the Rise

To date, the Robert Packard Center for ALS Research at Johns Hopkins has awarded 160 research grants and has several new grants pending, at a total of $29 million. Through an invitation-only process, the Center awards ALS research grants throughout the year on a flexible time frame, with the idea that rapid funding fosters innovative ideas.   Listed below is our current research portfolio.

Sami Barmada, MD, PhD
Mechanisms of Initiation and Progression in ALS

Don Cleveland, PhD
Identifying the Molecular Mechanism of Therapeutic Uptake of Antisense Oligonucleotides, Using a Genome-Scale CRISPR-Cas9 Mediated Gene Activation Strategy

Sandrine Da Cruz, PhD
Screening for Therapeutic Agents that Promote Muscle Innervation or Inhibit Denervation in ALS

Steven Finkbeiner, MD, PhD
Human Neuromuscular Models of ALS

Fen-Biao Gao, PhD
iPSC-Derived Motor Neuron Models of ALS with C9ORF72 Repeat Expansion

Aaron D. Gitler, PhD & Julianne Grose, PhD
Characterizing a Kinase Regulator of Ataxin-2 as a Therapeutic Target for ALS

Paschalis Kratsios, PhD
Molecular mechanisms that maintain motor neuron function throughout life

John Landers
Genome-Wide Screening of RNAi Towards Developing a Therapeutic for ALS

John Landers
Investigating Pridopidine, a Sigma-1 receptor activator, as a novel therapeutic treatment for ALS

Albert La Spada, MD, PhD
Role of Senataxin in Regulating c9orf72 Repeat Mediated Transcription Dysregulation and Neurotoxicity in ALS

Gabsang Lee, PhD, DVM & Nicholas Maragakis, MD
Derivation of Oculomotor Neurons using PHOX2b::GPF Reporter ALS-hiPSC Lines

Thomas Lloyd, MD, PhD
Axonal Transport and Retrograde Signaling in Drosophila Models of c9-ALS

Nicholas Maragakis, MD
Investigating the Contributions of Astrocyte Gap Junctions to ALS Disease Progression

Mervyn J. Monteiro, PhD
Generation and Characterization of Ubiquilin-2 Transgenic Mice Models of ALS

Mervyn J. Monteiro, PhD (Project co-funded with ALSA)
Gene Profiling to Determine the Initiator Mechanisms Involved in Disease in a UBQLN2 Mouse Model of ALS-FTD

Udai Pandey, PhD
Investigating the Molecular Mechanisms of FUS-Related ALS

Leonard Petrucelli, PhD
The Role of Epigenetic Changes and Downstream Pathological Events in c9FTD/ALS

Richard Robitaille, PhD
Restoring In Vivo Glial Functions at Neuromuscular Junctions in ALS: A New Therapeutic Target.

Rita Sattler, PhD
Role of Synaptic Dysfunction in C9orf72-Mediated pathogenesis in Patient-Derived iPS Neurons and in Vivo Animal Models

Shan Sockanathan, PhD
Mechanisms of Motor Neuron Loss in ALS

J. Paul Taylor, MD, PhD
Altered RNA Metabolism in C9ORF72-associated ALS

Peter Todd, MD, PhD
The Integrated Stress Response in ALS

Davide Trotti, PhD & Ya-Ming Hou, PhD
Deciphering RAN Translation and tRNA Requirements in C9orf72-ALS/FTD Tractable in Vitro Models

Marka van Blitterswijk, MD, PhD
Identification of novel biomarkers for C9ORF72-linked diseases, combining a targeted approach with an unbiased screen

Jiou Wang, MD, PhD
Using C. Elegans to Investigate Molecular Mechanisms of ALS

Jiou Wang, MD, PhD
New Pathways and Targets on RNA Homeostasis in ALS

Philip Wong, PhD
Validation of a Therapeutic Strategy to Repress TDP-43 Cryptic Exons for ALS

Philip Wong, PhD & Liam Chen, MD, PhD
Targeting TDP-43 Repression of Nonconserved Cryptic Exons in ALS-FTD

Zuoshang Xu, PhD
How Interneuron Expression of Mutant PFN1 Drives Motor Neuron Degeneration and Clinical Progression of ALS