CIRM's 1,000th published paper targets Huntington's disease

CIRM recently realized a noteworthy milestone with the publication of the 1,000th CIRM-funded paper in early December (here is a link to that paper). This is exciting to me personally because one of my first assignments when I joined CIRM three years ago was to develop (in collaboration with my Science Office colleague Rahul Thakar) a system and database for tracking CIRM-funded publications. At that point, there were only around 100 CIRM-funded papers, mostly from training grants, which were the first grants CIRM awarded in the spring of 2006. Watching the database grow and the publication rate accelerate has been exciting because these papers describe real progress that CIRM grantees are making in understanding stem cell biology, mechanisms of human disease and advancing stem cell-based therapies to the clinic.

The 1,000th paper itself is a great example of this progress. It was published in the journal Molecular & Cellular Neuroscience by Drs. Scott Olson, Jan Nolta and colleagues at UC Davis with the title “Examination of mesenchymal stem cell-mediated RNAi transfer to Huntington's disease affected neuronal cells for reduction of huntingtin”. The fact that the thousandth journal article comes from Jan Nolta's lab has added significance given that she was just yesterday named editor of one of the leading specialty journals in the field Stem Cells.

The promising research described in this paper was funded in large part by a CIRM Early Translational grant to Nolta, which has the goal of developing a combination cell and gene therapy candidate to treat Huntington’s Disease, a devastating neurological disease for which there is no effective treatment. Among those who have suffered and died from Huntington’s is one of my favorite songwriters, Woody Guthrie, whose 100th birthday will be celebrated in July. There's more information about Huntington's disease and awards we've funded available on our website.

In Huntington’s Disease, a mutated form of a protein called huntingtin causes certain neurons in the brain to die. The goal of Nolta’s award is to use stem cells to disable the ability of those neurons to make the disease-causing protein. In their paper, Nolta and colleagues show that a type of stem cell found in the bone marrow called mesenchymal stem cells (MSCs) can be engineered to produce a molecule that inhibits the production of the mutated huntingtin protein. They show, in a petri dish, that this molecule is secreted by the stem cells, is taken up by nearby neuronal cells, and reduces the amount of the disease-causing protein produced by those cells. The next important step will be testing these engineered stem cells in an animal model of Huntington’s.

With over a thousand CIRM-funded publications come a lot of data and statistics that allow us to assess our programs and progress. Of the 1,009 CIRM-funded publications, 770 describe original research, 175 are literature reviews and 64 describe novel scientific methods. The top four RFAs by publication number are, not surprisingly, the four earliest in CIRM’s funding history: Training grants with 574 publications, New Faculty with 178, Comprehensive with 169 and SEED with 160. CIRM funding has resulted in an impressive number of papers in elite journals: 55 in Nature, 47 in Cell Stem Cell, 24 in Cell and 13 in Science. In total,298 CIRM-funded papers have been published in journals with impact factors greater than 10. There were 10 CIRM-funded publications in 2006, 68 in 2007, 151 in 2008, 224 in 2009, 261 in 2010, and 295 in 2011, so I predict that I’ll be back to blog about the 2,000th paper before the end of 2014!

Zachary Scheiner is a Science Officer at CIRM


Olson SD, Kambal A, Pollock K, Mitchell GM, Stewart H, Kalomoiris S, Cary W, Nacey C, Pepper K, & Nolta JA (2011). Examination of mesenchymal stem cell-mediated RNAi transfer to Huntington's disease affected neuronal cells for reduction of huntingtin. Molecular and cellular neurosciences PMID: 22198539