School of Medicine


Showing 1-20 of 48 Results

  • Rajni Agarwal-Hashmi

    Rajni Agarwal-Hashmi

    Associate Professor of Pediatrics (Stem Cell Transplantation) at the Lucile Salter Packard Children's Hospital

    Current Research and Scholarly Interests Hematopoietic Stem cell biology-created a SCID mouse model to study engraftment of cord blood derived hematopoietic cells and use of this model to develop gene transfer technology for Fanconi anemia.
    Clinical research interests are to develop new protocols to reduce graft vs host disease,treatment of viral infections post transplant and use of manipulated HSC graft in patients who receive mismatched donor transplants.

  • Michael Amylon

    Michael Amylon

    Professor of Pediatrics (Hematology/Oncology) at the Lucile Salter Packard Children's Hospital, Emeritus

    Current Research and Scholarly Interests Bone marrow transplantation (BMT) is a treatment modality which is being broadly applied to a growing number of disorders. Increasing success with BMT is offering improved survival to pediatric and adult patients with acute leukemia, chronic leukemia, lymphomas, and a variety of solid tumors as well as severe aplastic anemia.

  • Rosa Bacchetta

    Rosa Bacchetta

    Associate Professor (Research) of Pediatrics (Stem Cell Transplantation)

    Current Research and Scholarly Interests In the coming years, I plan to further determine the genetic and immunological basis of diseases with autoimmunity or immune dysregulation in children. I believe that much can still be learned from the in depth mechanistic studies of pediatric autoimmune diseases. Genomic analysis of the patients' samples has become possible which may provide a rapid indication of altered target molecules. I plan to implement robust functional studies to define the consequences of these genetic abnormalities and bridge them to the patient's clinical phenotype.

    Understanding functional consequences of gene mutations in single case/family first and then validating the molecular and cellular defects in other patients with similar phenotypes, will anticipate and complement cellular and gene therapy strategies.

    For further information please visit the Bacchetta Lab website:
    http://med.stanford.edu/bacchettalab.html

  • Alice Bertaina

    Alice Bertaina

    Associate Professor of Pediatrics (Stem Cell Transplantation) at the Lucile Salter Packard Children's Hospital

    Current Research and Scholarly Interests Dr. Bertaina is a highly experienced clinician and will play a key role in supporting Section Chief Dr. Rajni Agarwal and Clinical Staff in the Stem Cell Transplant Unit at Lucile Packard Children’s Hospital. She will also continue her research on immune recovery and miRNA, understanding the mechanisms underlying immune reconstitution, Graft-versus-Host Disease (GvHD), and leukemia relapse after allogeneic HSCT in pediatric patients affected by hematological malignant and non-malignant disorders.

  • Alma-Martina Cepika

    Alma-Martina Cepika

    Senior Research Scientist, Pediatrics - Ped Stem Cell Transplantation

    Current Role at Stanford As a senior research scientist in the laboratory of Dr. Maria Grazia Roncarolo, Dr. Cepika is working to identify a molecular landscape of inducible type 1 regulatory T cells (Tr1), and how it defines their functions. Tr1 designed to be tolerant specifically to patient tissue are currently investigated in a phase I clinical trial, with an ultimate goal to use Tr1 cell therapy to prevent graft-vs-host disease in patients with hematological cancers undergoing allogeneic hematopoietic stem cell transplantation (NCT03198234). Identifying the components critical for identity of these cells and understanding their mechanism of action is an essential step for harnessing their suppressive power in the clinic.

  • Agnieszka Czechowicz

    Agnieszka Czechowicz

    Assistant Professor of Pediatrics (Stem Cell Transplantation)

    Current Research and Scholarly Interests Dr. Czechowicz’s research is aimed at understanding how hematopoietic stem cells interact with their microenvironment in order to subsequently modulate these interactions to improve bone marrow transplantation and unlock biological secrets that further enable regenerative medicine broadly. This work can be applied across a variety of disease states ranging from rare genetic diseases, autoimmune diseases, solid organ transplantation, microbiome-augmentation and cancer.

  • Daniel Dever

    Daniel Dever

    Instructor, Pediatrics - Stem Cell Transplantation

    Bio Dr. Daniel Dever is a Research Instructor in the laboratory of Dr. Matthew Porteus at Stanford University, in the Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine. He completed his PhD in molecular toxicology at the University of Rochester where he studied the mechanisms of the aryl hydrocarbon receptor in mediating cerebellar transcriptional programs. During his postdoctoral work in the Porteus group, he (with others) developed a CRISPR/Cas9-based beta-globin (HBB) gene editing by homologous recombination methodology (gene targeting) in CD34+ hematopoietic stem cells as a potential therapeutic strategy to treat severe sickle cell disease. Dr. Dever (along with collaborators) has now successfully used this methodology to efficiently target >15 genes in primary blood cells that are associated with hematopoiesis, hematopoietic genetic diseases, hematopoietic malignancies, or safe harbor sites. Dr. Dever's primary research interests are to continue to leverage CRISPR/Cas9-based genome editing technologies to study the molecular mechanisms of gene targeting in human hematopoietic stem cells with the ultimate goal of optimizing and further developing novel cell and gene therapies for disease of the blood and the immune system. Currently, he is leading IND-enabling preclinical efficacy, feasibility, safety and tumorigenicity studies for FDA approval of a first-in-human clinical trial at Stanford in 2018 for the treatment of severe sickle cell disease using CRISPR/Cas9-based HBB gene targeting in autologous hematopoietic stem cells.

  • M Carolina Gallego Iradi

    M Carolina Gallego Iradi

    Thymus Research Assistant, Pediatrics - Ped Stem Cell Transplantation

    Bio • In October 2017 my previous research related to Alzheimer's signs in dolphins got worldwide recognition appearing in tv and newspapers from all around the world such Newsweek, The Times, CBS, Discover, The Chicago Post, Los Angeles Times, Nature, National Geographic Italy (2018) and more. I recently joined to Stanford University (Stem Cell Transplantation and Regenerative Medicine).

    • My initial training focused on marine biology, with special emphasis on trace metal pollution. This work, which involved collaborations with different governmental, private (as Venezuelan Oil Company) and University entities (national and international), was well recognized in Venezuela where I received a merit award by the Town Hall of Porlamar (Margarita Island).

    • After graduation, I obtained a fellowship to enroll in a Master Degree program in Waste Management at the Universidad Internacional de Andalucia (Spain). My performance in this program allowed me to obtain a very prestigious Biomedicine fellowship (one awarded in all of Latin America) from the Santander-Central-Hispano Bank to begin a PhD in Genetics and Development related to Neuroscience at Universidad de Zaragoza (Spain).

    • My PhD studies focused on comparative pathology, demonstrating pathology similar to Alzheimer's Disease in dogs and cetaceans. After a few years of work in academia in Venezuela, I obtained an opportunity to become a post-doctoral fellow at the University of Florida (USA).

    • In 2014, I transferred to the Department of Neuroscience to work with Drs. David Borchelt. Since this time, my work has focused on understanding how mutations in Matrin 3 cause ALS and myopathy. In 2016, I was promoted to an entry level faculty position (Assistant Scientist) in the Department of Neuroscience and my goal includeded the use of techniques in genetics,, cellular biology and chemistry to develop a research program that spans basic science to pharmacological application in neuromuscular disorders.

  • Natalia Gomez-Ospina

    Natalia Gomez-Ospina

    Assistant Professor of Pediatrics (Genetics) and of Pediatrics (Stem Cell Transplantation)

    Current Research and Scholarly Interests Dr. Gomez-Ospina is a physician scientist and medical geneticist with a strong interest in the diagnosis and management of genetic diseases.

    1) Lysosomal storage diseases:
    Her research program is on developing better therapies for a large class of neurodegenerative diseases in children known as lysosomal storage disorders. Her current focus is on developing genome editing of hematopoietic stem cells as a therapeutic approach for these diseases beginning with Mucopolysaccharidosis type 1 and Gaucher disease. She established a genetic approach where therapeutic proteins can be targeted to a single well-characterized place in the genome known as a safe harbor. This approach constitutes a flexible, “one size fits all” approach that is independent of specific genes and mutations. This strategy, in which the hematopoietic system is commandeered to express and deliver therapeutic proteins to the brain can potentially change the current approaches to treating childhood neurodegenerative diseases and pave the way for alternative therapies for adult neurodegenerative disorders such as Alzheimer’s and Parkinson’s disease


    2) Point of care ammonia testing
    She also works in collaboration with other researchers at Stanford to develop point-of-care testing for serum ammonia levels. Such device will greatly improve the quality of life of children and families with metabolic disorders with hyperammonemia.

    3) Gene discovery
    Dr Gomez-Ospina lead a multi-institutional collaboration resulting in the discovery of a novel genetic cause of neonatal and infantile cholestatic liver disease. She collaborated in the description of two novel neurologic syndromes caused by mutations in DYRK1 and CHD4.