Researches Archive - Vision2030

Research Portfolio

Repair and Regeneration Research

Brain-Computer Interfaces

Leigh Hochberg, MD, PhD

There is an urgent need to ensure people with ALS will never lose the ability to communicate. Led by Leigh Hochberg, MD, PhD and the extraordinary BrainGate team at Mass General, together with an unmatched multi-institutional and multidisciplinary team, we are getting closer to achieving that goal.

Discovering subtypes of ALS

Aneesh Donde, PhD

When the root cause of ALS is known, there are now paths to therapy, as has been shown by the recent development of tofersen for people whose disease results from the SOD1 gene.

Investigating Therapeutic Options to Increase STMN2 Levels

Clotilde Lagier-Tourenne, MD, PhD

Researchers at the Sean M. Healey & AMG Center for ALS, along with their colleagues, demonstrated that pharmacological compounds and gene therapy that increase the levels of STMN2 can restore the ability of neurons to grow axons after injury.

Using Stem Cells to Improve Motor Function

Brian Wainger, MD, PhD

Brian Wainger, MD, PhD, is developing a novel therapy to help improve motor outcomes in nerve injury and ALS in collaboration with Justin Brown, MD in the Department of Neurosurgery. The strategy is to transplant engineered motor neurons that have been derived in the laboratory from human stem cells.

The Impact of Gene Fusions on ALS Disease Progression

Ghazaleh Sadri-Vakili, MD, PhD

We predict that ALS-specific gene fusions may improve many aspects of clinical care as already demonstrated in the field of oncology and are now working to understand how the fusions effect ALSdisease progression.

Identifying novel therapeutic targets related to TDP-43 loss

Velina Kozareva

Despite the diversity of ALS, almost all patients have one thing in common: the protein TDP-43 stops performing its normal functions in the nucleus, leading to disrupted splicing and decreased expression of many genes.

Wainger Therapeutic proposal

ALS and Frontotemporal Disorder are likely caused by multiple pathological processes occurring at different locations on motor neurons.

All research

Address the
root cause

Discover
novel therapies

Enhance
neuroplasticity

Implement
neurotechnology

Pioneer disruptive
approaches