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Researchers bring gene editing to patients with deadly

Maria-Grazia Roncarolo of San Raffaele Scientific Institute, Milan (OSR) with expertise in: Pediatrics, Hematology and Immunology. OSR · Division of Regenerative Medicine, Stem Cells and Gene

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XRCC1 mutation is associated with PARP1 hyperactivation

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Leading stem-cell expert to join Stanford Medicine faculty

A complex combination of genetic and environmental factors is known to lead to the development of type 2 diabetes. Obesity is the leading risk factor for the condition, which is brought on by overeating.

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The gene therapy will be administered without conditioning chemotherapy, and will require a (relatively) short hospital stay to collect stem cells over an approximate 6-day period, followed by a single intravenous administration of gene-corrected hematopoietic cells over the course of one hour or less.

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GSK, Fondazione Telethon and Fondazione San Raffaele to

A single sperm was placed into a single egg to create an embryo. Then the gene editing tool was added. When the embryos were 3 to 5 days old, a few cells were removed and checked for editing.

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Vasseur, F. , Helbecque, N. , Dina, C. , Lobbens, S. , Delannoy, V. , Gaget, S. et al. (2002) Single-nucleotide polymorphism haplotypes in the both proximal promoter and exon 3 of the APM1 gene modulate adipocyte-secreted adiponectin hormone levels and contribute to the genetic risk for type 2 diabetes in French Caucasians.

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Emergence of Linezolid Resistance in the Vancomycin

3/9/2014 · Premature Ovarian Failure: A Critical Condition in The Reproductive Potential with Various Genetic Causes Farkhondeh Pouresmaeili , Ph.D 1, 2, * and Zahra Fazeli , M.Sc 1 1 Department of Medical Genetics, Faculty of Medicine, Shahid Beheshti University of Medical Sciences, Tehran, Iran

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12/21/2016 · XRCC1 is a molecular scaffold protein that assembles multi-protein complexes involved in DNA single-strand break The gene mutated in ataxia-ocular apraxia 1 …

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Adiponectin gene polymorphisms and their effect on the

Linezolid-resistant isolates carried the G2576T mutation in the 23S rRNA gene, belonged to different clones, and shared the same allelic profile, which clusters in the C1 multilocus sequence typing epidemic lineage. Maria Grazia Bonora, Maurizio Solbiati, Erminia Stepan, Antonella Zorzi, The molecular basis of linezolid resistance has

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Correction of ADA-SCID by Stem Cell Gene Therapy Combined

Jean Genet (French: [ʒɑ̃ ʒənɛ]; () 19 December 1910 – () 15 April 1986) was a French novelist, playwright, poet, essayist, and political activist. Early in his life he was a …

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WASHINGTON: Researchers have identified a single variation in a gene that influences obesity in children and adults by lowering the levels of a certain protein that helps us feel full.

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Lentiviral haemopoietic stem-cell gene therapy in early

5/6/2016 · A gene therapy for an inherited immune disease completes a 27-year journey. and many aspire to be out-and-out cures for one of about 5,000 rare diseases caused by errors in a single gene

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Premature Ovarian Failure: A Critical Condition in The

Cancer genetics and genomics of human FOX family genes. Single nucleotide polymorphism (SNP) within the 5′-UTR of the FOXE1 I. Venza, M. Visalli, B. Tripodo, G. de Grazia, S. Loddo, D. Teti, M. VenzaFOXE1 is a target for aberrant methylation in cutaneous squamous cell carcinoma.

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Grazia Graziani | University of Rome Tor Vergata, Rome

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Gene Therapy 2.0: 10 Breakthrough Technologies 2017 - MIT

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Single gene variation linked to obesity - Times of India

GSK, Fondazione Telethon and Fondazione San Raffaele to collaborate on gene therapy for rare diseases 18 October 2010 This press release is intended for business journalists and analysts/investors.

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Roncarolo | Institute for Stem Cell Biology and

Researchers bring gene editing to patients with deadly diseases More than 10,000 human diseases are caused by a single gene defect, according to Dr. Maria Grazia Roncarolo, co-director of

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2/22/2017 · Researchers are studying gene therapy in clinical trials for about 40 to 50 different diseases, says Maria-Grazia Roncarolo, a pediatrician and scientist at Stanford University who led early gene

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Researchers uncover single gene associated with type 2

Leading stem-cell expert to join Stanford Medicine faculty. Maria Grazia Roncarolo, MD, a stem cell and gene therapy expert and former scientific director of the “No single person has done as much as she in this field, or as successfully,” said

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Maria Grazia Biferi, 1Mathilde Cohen-Tannoudji, Ambra Cappelletto, 1Benoit Giroux, a single intravascular injection.10 This systemic AAV9 approach was 2038 Molecular Therapy Vol. 25 No 9 September 2017 ª 2017 The American Society of Gene and Cell Therapy. the administration of ASOs against mutant human SOD1 (hSOD1)

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Grazia Graziani of University of Rome Tor Vergata, Rome (UNIROMA2) with expertise in: Pharmacy, Cancer Research and Molecular Biology. UNIROMA2 · Dipartimento di Medicina dei Sistemi. 41.86

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A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and

12/14/2014 · A nsSNP in the bovine TLR2 gene (Val220Met) has been shown to decrease the response to M. avium subsp. paratuberculosis (Map) , while another SNP in the same gene, C1903T (Silent569), was found to be associated with resistance to Map infection in cattle .

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First gene-edited babies claimed in China - Times of India

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6/23/1998 · Familial multiple system tauopathy with presenile dementia (MSTD) is a neurodegenerative disease with an abundant filamentous tau protein pathology. Multiple system tauopathy with presenile dementia (MSTD) They range from 352 to 441 amino acids and are produced from a single gene by alternative mRNA splicing. They contain

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Mutation in the tau gene in familial multiple system

Maria Grazia Roncarolo's lab leads efforts to translate scientific discoveries in genetic diseases and regenerative medicine into novel patient therapies, including treatments based on stem cells and gene therapy.Dr. Roncarolo, a pediatric immunologist by training, spent her early career in Lyon

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Detection of BRAF gene mutation in primary choroidal

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Maria Grazia Roncarolo is part of Stanford Profiles, official site for faculty, postdocs, students and staff information (Expertise, Bio, Research, Publications, and more). are caused by single-gene mutations resulting in immune deficiency and dysregulation. More than 350 diseases have been described to date, and the number is rapidly

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Gene Therapy’s First Out-and-Out Cure Is Here - MIT

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Cancer genetics and genomics of human FOX family genes

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