CHORI Staff Directory
CHORI Intranet

 

CHORI CURRENT News

2017

The Vitamin Lipoic Acid Prevents Kidney Stones in Mouse Model of a Rare Genetic Disease
Cystinuria is caused by a mutation in a protein that transports the amino acid cysteine in the kidney and GI. The main clinical impact of the disease in a build up of cysteine in the urine, which then aggregates and forms painful kidney stones in children and adults. Stone formation can be quite rapid and frequent, leading to a poor quality of life. Few medications exist for these patients, and all have significant side effects. In a study conducted by The Buck Institute for Research on Aging and UCSF Department of Urology, high doses of the natural vitamin lipoic acid was found to shutdown cystine stone formation in mice with the same genetic defect as found in human cystinuria. Moreover, the benefits of lipoic acid happened quickly and without any detectable side effects (other than smelly urine). The UCSF clinical team is now recruiting for a clinical trial to determine if lipoic acid will be effective in cystinuria patients. UCSF Benioff Children's Hospital Oakland Research Institute scientist David Killilea, with joint appointment in the UCSF Department of Urology, contributed to this study.

Click here to download the complete press release.
Click here to download the article.
Clinical study: https://clinicaltrials.gov/ct2/show/NCT02910531

CHORI Scientist receives $100,000 grant from Abate Med to help find a lasting treatment for Atopic Dermatitis
Laura Hertel, PhD, a virologist at Children’s Hospital Oakland Research Institute (CHORI), has received a grant from Abate Med’s founder, Dr. David Paslin, for a pre-clinical study on the efficacy of using a protein made by a common virus that infects the skin, to treat the cutaneous manifestations of atopic dermatitis.
Click here to download the complete press release.

2016

Researchers Discover a New Gatekeeper Role for Thymic Dendritic Cells in Controlling T Cell Release into the Bloodstream: Better Understanding of Cell's Role Could Lead to New Strategies to Treat Autoimmune Diseases, Cancer
A team of scientists led by Julie Saba, MD, PhD at UCSF Benioff Children’s Hospital has unveiled a novel role of thymic dendritic cells, which could result in new strategies to treat conditions such as autoimmune diseases, immune deficiencies, prematurity, infections, cancer, and the loss of immunity after bone marrow transplantation.

The study “Dendritic cell sphingosine-1-phosphate lyase regulates thymic egress,” appeared in the ‘Journal of Experimental Medicine’ (November 14, 2016 issue), published by Rockefeller University Press (http://jem.rupress.org/content/213/12/2773).
Click here to download the complete press release.

CHORI Research on Genome Editing for a Sickle Cell Cure Receives nearly $4.5 million from the California Institute for Regenerative Medicine
UCSF Benioff Children's Hospital Oakland's Dr. Mark Walters has received a nearly $4.5 million grant from the California Institute for Regenerative Medicine (CIRM) for his research project, "Curing Sickle Cell Disease with CRISPR-Cas9 genome editing." It is one of four research projects, focused on diseases that have an enormous impact on life expectancy and quality of life, that have been awarded a total of $15 million by the governing Board of CIRM.

The funding is part of the Translational Award program, which has a goal of moving the most promising projects out of the laboratory and into clinical trials in people.

Dr. Walters' research uses a genetic editing tool, CRISPR-Cas9, to help find a cure for sickle cell disease. The research involves taking the patient’s own blood stem cells, using CRISPR-Cas9 to correct the genetic mutation causing the disease, and then return those cells to the patient. It’s hoped the “corrected” blood stem cells will then multiply and create a new blood system, one free of sickle cell. The funding from CIRM will enable Dr. Walters' team to do the preliminary testing and research needed to get this project ready for a clinical trial.
Click here to download the complete press release.

UCSF Benioff Children’s Hospitals and Partners to Receive $1.2 Million Grant from California Initiative to Advance Precision Medicine
A team at the Oakland and San Francisco campuses of UCSF Benioff Children’s Hospitals, UCSF, and UC Berkeley have been awarded $1.2 million by California Initiative to Advance Precision Medicine (CIAPM) to help advance precision medicine in the state.

Major children’s hospitals such as the UCSF Benioff Children’s Hospitals often see patients who have unusual and severe inborn conditions that appear to be genetic, but the gene responsible for the disorder is unknown. The CIAPM-supported project, “Full Genome Analysis of Children to Guide Precision Medicine” led by Dr. David Martin of the Children’s Hospital Oakland Research Institute (CHORI), will help advance precision medicine by developing methods that improve our ability to identify mutations that cause inherited diseases, and to find the cause of such difficult-to-diagnose genetic conditions.
Click here to download the complete press release.

Genome engineering paves the way for sickle cell cure: Repairing the sickle cell mutation in stem cells holds promise for new treatment
A team of physicians and laboratory scientists has taken a key step toward a cure for sickle cell disease, using CRISPR-Cas9 gene editing to fix the mutated gene responsible for the disease in stem cells from the blood of affected patients. For the first time, they have corrected the mutation in a proportion of stem cells that is high enough to produce a substantial benefit in sickle cell patients.

The researchers from UC San Francisco Benioff Children’s Hospital Oakland Research Institute (CHORI), the University of California, Berkeley and the University of Utah School of Medicine hope to re-infuse patients with the edited stem cells and alleviate symptoms of the disease, which primarily afflicts those of African descent and leads to anemia, painful blood blockages and early death.

In tests in mice, the genetically engineered stem cells stuck around for at least four months after transplantation, an important benchmark to ensure that any potential therapy would be lasting.

“This is an important advance because for the first time we show a level of correction in stem cells that should be sufficient for a clinical benefit in persons with sickle cell anemia,” said co-author Mark Walters, a pediatric hematologist and oncologist and director of UCSF Benioff Oakland’s Blood and Marrow Transplantation Program.

The results will be reported in the Oct. 12, 2016 issue of the online journal Science Translational Medicine (http://stm.sciencemag.org/content/8/360/360ra134).
Click here to download the complete press release.

Ellen Fung, PhD RD (CHORI) and her colleagues Laura Bachrach, MD (Stanford) and Aenor Sawyer, MD (UCSF) just published the 2nd edition of the text, Bone Health Assessment in Pediatrics: Guidelines for Clinical Practice (Springer, 2016). The text is the gold-standard resource for evaluating bone health in children and adolescents. Authors include an international panel of experts in measuring and analyzing bone density in the pediatric patient, and chapters reflect the most recent International Society for Clinical Densitometry (ISCD) Guidelines for Pediatric DXA assessment, interpretation and reporting. This indispensable reference covers all the important changes in the field over the last decade. Some highlights of this edition include: an entire chapter on the assessment of infants and toddlers, a chapter devoted to the assessment of children with disabling conditions, an in-depth discussion of vertebral fracture and its etiologies, and a thorough review of the advantages and limitations of densitometry techniques including DXA, pQCT, HRpQCT, and MRI. Solidifying itself as the leading text in the field, Bone Health Assessment in Pediatrics: Guidelines for Clinical Practice, 2nd ed. provides all of the critical basic analysis and evaluation tools, images, and calculations necessary for both researchers as well as practicing clinicians.

CHORI Study Reveals Potential Improvements for Effectiveness of Meningococcal Vaccines
A study conducted by UCSF Benioff Children's Hospital Oakland Research Institute (CHORI) scientists shows greatly improved protective antibody responses to a new mutant vaccine antigen for prevention of disease caused by Neisseria meningitidis - also known as meningococcus - that has the potential to improve the current vaccines for meningitis. 

The study, “Enhanced Protective Antibody to a Mutant Meningococcal Factor H-Binding Protein with Low Factor H Binding,” authored by Children’s Hospital Oakland Research Institute (CHORI) scientists Dan Granoff, MD, and Peter Beernink, PhD was featured in the September 8th, 2016 issue of the Journal of Clinical Investigation Insight.

The UCSF Benioff Children’s Hospital Oakland press release is located at http://174.143.136.176/Main/News/365.aspx and the original article is available at https://insight.jci.org/articles/view/88907

SJND Students Intern at Stanford, Children’s Hospital Oakland Labs
Anna Victoria Serbin ’17 was selected for the 2016 CHORI (Children’s Hospital Oakland Research Institute) summer research internship. She was one of 12 students selected from a very competitive pool of more than 1,000 applicants.

35th Annual CHORI Summer Student Research Symposium:
A Golden Affair


On August 12, 2016 the CHORI community hosted its’ 35th Summer Student Research Symposium. Enthusiastic students, proud parents and family members, CHORI & UCSF Benioff Children’s Hospital faculty mentors and staff, and many friends joined together to celebrate the accomplishments of this summers’ class. During the summer, 44 students (64% female, 34% in high school, 77% under-represented minority, low income or with a disability) worked one-on-one with their research mentor to develop their own unique hypothesis driven project. Mentors were recruited not only from the CHORI and BCHO campus, but also from the UC Berkeley Stem Cell Center, and the UCSF Parnassus, Laurel Heights and SF General Campuses. This broad range of mentors allowed for a very diverse research experience for the students.

 



Thursday, March 9, 2017 11:11 AM

© 2005 Children's Hospital Oakland Research Institute
5700 Martin Luther King Jr Way • Oakland, California 94609
Phone 510-450-7600 • Fax 510-450-7910
Site MapDisclaimer