Marisa Cappella

Post-doctoral researcher

Marisa Cappella

Titles and positions

PhD, Post-Doctoral researcher


  • Gene therapy
  • Gene editing (CRISPR-Cas9)
  • Repeat expansions diseases


After a Master Degree in “Medical Biotechnology” I obtained a PhD in “Morphogenesis and Tissue Engineering” at the University of Rome “La Sapienza” in 2018. During these years, I worked in the field of dementia and skeletal muscle pathology. In particular I gained experience in the research of molecular biomarkers, in the field of gene expression mediated by microRNAs and of gene editing. In particular, during my training for Master's degree, I participated to the identification of blood biomarkers in Alzheimer's dementia and I studied microRNAs expression in response to hypoxia in an in vitro glioblastoma model. During my PhD studies, I characterized miRNAs involved in skeletal muscle differentiation and I generated CRISPR/Cas9 tools to excise the repeat expansions that causes Myotonic Dystrophy type 1. With this background and knowledge on disease modifying approaches, I joined the group of Dr. Biferi for a post-doctorate, in February 2018. I work on the development of efficient and innovative gene therapy strategies for Amyotrophic Lateral Sclerosis (ALS), a fatal motor neuron (MN) disorder, with a particular interest in familial ALS caused by C9orf72 mutations.

Main publications

  1. Mallozzi, C, Crestini, A, D'Amore, C, Piscopo, P, Cappella, M, Perrone, F et al.. Activation of Tyrosine Phosphorylation Signaling in Erythrocytes of Patients with Alzheimer's Disease. Neuroscience. 2020;433 :36-41. doi: 10.1016/j.neuroscience.2020.02.050. PubMed PMID:32156551 .
  2. Cappella, M, Ciotti, C, Cohen-Tannoudji, M, Biferi, MG. Gene Therapy for ALS-A Perspective. Int J Mol Sci. 2019;20 (18):. doi: 10.3390/ijms20184388. PubMed PMID:31500113 PubMed Central PMC6771059.
  3. Cappella, M, Perfetti, A, Cardinali, B, Garcia-Manteiga, JM, Carrara, M, Provenzano, C et al.. High-throughput analysis of the RNA-induced silencing complex in myotonic dystrophy type 1 patients identifies the dysregulation of miR-29c and its target ASB2. Cell Death Dis. 2018;9 (7):729. doi: 10.1038/s41419-018-0769-5. PubMed PMID:29955039 PubMed Central PMC6023919.
  4. Provenzano, C, Cappella, M, Valaperta, R, Cardani, R, Meola, G, Martelli, F et al.. CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients. Mol Ther Nucleic Acids. 2017;9 :337-348. doi: 10.1016/j.omtn.2017.10.006. PubMed PMID:29246312 PubMed Central PMC5684470.
  5. Cardinali, B, Cappella, M, Provenzano, C, Garcia-Manteiga, JM, Lazarevic, D, Cittaro, D et al.. MicroRNA-222 regulates muscle alternative splicing through Rbm24 during differentiation of skeletal muscle cells. Cell Death Dis. 2016;7 (2):e2086. doi: 10.1038/cddis.2016.10. PubMed PMID:26844700 PubMed Central PMC4849150.
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