CellR4-- repair, replacement, regeneration, & reprogramming最新文献

{"title":"Transplanting islet cells can fix brittle diabetes. Why isn't it available in the U.S.?","authors":"C Ricordi,&nbsp;A Japour","doi":"10.32113/cellr4_201910_2768","DOIUrl":"https://doi.org/10.32113/cellr4_201910_2768","url":null,"abstract":"<p><p>Type 1 diabetes, which affects 1.25 million American children and adults, and more than 20 million people around the world, is a challenging chronic disease caused by the body's inability to make insulin. Among its most severe forms is brittle diabetes. People with brittle diabetes frequently experience large swings in blood sugar that can quickly move from too high to too low or vice versa. Severely low blood sugar, called hypoglycemia, can cause sudden and unexpected seizures, coma, heart attacks, and even death. Insulin is the main treatment for this common disease. But it isn't a cure. A type of cell transplant that comes close to a cure for some people with type 1 diabetes, a technique pioneered and tested in the United States, is now available in many countries but is still deemed an experimental procedure in the U.S., making it almost impossible to get. More than a decade ago, the United Kingdom's National Health Service approved islet cell transplantation for type 1 diabetes - an approval based on an extensive review of the evidence generated by clinical trials conducted in the United States. Our federal dollars supported that research, and this treatment ought to be available to U.S. citizens. Islet cell transplantation is not a panacea for all forms of type 1 diabetes. And transplantation of any organ, including islet cells, requires the use of anti-rejection drugs that can have a range of adverse side effects. Nevertheless, individuals with severe brittle diabetes who are fully informed of the risks and benefits should have the ability to access this lifesaving treatment option. We fully understand the FDA's efforts to rein in companies marketing unapproved stem cell products that have little or no evidence to support their use and that may put patients at risk. Yet the FDA should stay equally focused on its commitment to approving evidence-based transformative treatments for devastating diseases and conditions, including brittle diabetes.</p>","PeriodicalId":87230,"journal":{"name":"CellR4-- repair, replacement, regeneration, & reprogramming","volume":"7 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2019-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8025929/pdf/nihms-1057430.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25590188","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A case of a mild Wolfram Syndrome with concomitant <i>ATP7B</i> mutation.","authors":"R Squitti,&nbsp;G Cerchiaro,&nbsp;I Giovannoni,&nbsp;P Francalanci,&nbsp;M Siotto,&nbsp;P Maffei,&nbsp;C Ricordi,&nbsp;M C Rongioletti","doi":"10.32113/cellr4_20198_2735","DOIUrl":"https://doi.org/10.32113/cellr4_20198_2735","url":null,"abstract":"<p><strong>Background: </strong>Wolfram Syndrome 1 (WS1) has been characterized on the basis of mutation in the WFS1 gene encoding a calcium storage wolframin endoplasmatic reticulum transmembrane glycoprotein.</p><p><strong>Patients and methods: </strong>We observed a WS 10-years old female subject, with Type 1 diabetes-mellitus (DM), that had compound heterozygous WSF1 mutations but without other symptoms generally observed in WS subjects, such as optic atrophy or neurodegeneration.</p><p><strong>Results: </strong>Decreased copper, ceruloplasmin, and transferrin levels, pointing to a copper deficiency, were associated with a new c.18703A>G mutation in the ATP7B gene, while lower calcium levels were associated with WSF1 mutations. An omega-3 fatty acids therapy was administrated to the subject in the attempt to ameliorate diabetes symptoms, restored copper deficiency, and normal calcium levels.</p><p><strong>Conclusions: </strong>This specific case report provides new insights into the potential interplay of ATP7B mutation in shaping a milder WS clinical picture.</p>","PeriodicalId":87230,"journal":{"name":"CellR4-- repair, replacement, regeneration, & reprogramming","volume":"7 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2019-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8048209/pdf/nihms-1057379.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38885684","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Vitamin D and Omega 3 Field Study on Progression of Type 1 Diabetes.","authors":"C Ricordi,&nbsp;M Clare-Salzler,&nbsp;M Infante,&nbsp;C Baggerly,&nbsp;J Aliano,&nbsp;S McDonnell,&nbsp;S Chritton","doi":"10.32113/cellr4_20198_2737","DOIUrl":"https://doi.org/10.32113/cellr4_20198_2737","url":null,"abstract":"<p><p>Chronic inflammation has been linked to the progression of type 1 diabetes (T1D). Supplementation with vitamin D and omega-3 fatty acids, which have anti-inflammatory properties, may slow or stop the progression of T1D. A field study is underway to assess the relationship between these nutrients and T1D progression among auto-antibody positive individuals who have not been diagnosed with T1D. The T1D Prevention Field Study is currently recruiting participants to complete online health surveys and home blood-spot tests for 25-hydroxyvitamin D [25(OH)D], Omega-3 Index, AA:EPA Ratio, high-sensitivity C-reactive protein, and HbA1c every three to four months for 5 years. Participants (or their parents/guardians) are given information about the importance of achieving a 25(OH)D level between 40-60 ng/ml and an AA:EPA Ratio between 1.5-3.0 to reduce inflammation. However, participants are free to choose their own supplement or dietary regimens. Data analysis will focus on associations between vitamin D and omega-3 status and progression of T1D. Initial enrollment in the T1D Prevention Field Study includes 103 participants from fifteen countries; total enrollment is expected to reach at least 400 participants by the end of 2022. The field study approach allows for cost-effective research that capitalizes on new technologies for recruitment, data collection, and blood level testing from home. However, some challenges have arisen. Many individuals are reading the open source protocols and some choose to supplement and test on their own so incentives may be needed to increase enrollment. Additionally, some participants do not have access to auto-antibody testing or are unable to get access to their test results; therefore, there is a need to provide blood spot auto-antibody testing through the field study.</p>","PeriodicalId":87230,"journal":{"name":"CellR4-- repair, replacement, regeneration, & reprogramming","volume":"7 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2019-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6768421/pdf/nihms-1050723.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41223150","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Islet cell autotransplantation update.","authors":"Khawla F Ali,&nbsp;Betul Hatipoglu","doi":"","DOIUrl":"","url":null,"abstract":"","PeriodicalId":87230,"journal":{"name":"CellR4-- repair, replacement, regeneration, & reprogramming","volume":"7 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2019-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7059780/pdf/nihms-1066723.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37718547","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Can high-dose omega-3 fatty acids and high-dose vitamin D3 (cholecalciferol) prevent type 1 diabetes and sustain preservation of beta-cell function after disease onset?","authors":"C Ricordi,&nbsp;G Lanzoni","doi":"","DOIUrl":"","url":null,"abstract":"","PeriodicalId":87230,"journal":{"name":"CellR4-- repair, replacement, regeneration, & reprogramming","volume":"6 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2018-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8276914/pdf/nihms-1056746.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39185476","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"POSEIDON study: a pilot, safety and feasibility trial of high-dose omega3 fatty acids and high-dose cholecalciferol supplementation in type 1 diabetes.","authors":"D A Baidal,&nbsp;J Sanchez,&nbsp;R Alejandro,&nbsp;C E Blaschke,&nbsp;K Hirani,&nbsp;D L Matheson,&nbsp;S Messinger,&nbsp;A Pugliese,&nbsp;L E Rafkin,&nbsp;L A Roque,&nbsp;J M Vera Ortiz,&nbsp;C Ricordi","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>The anti-inflammatory and immunomodulatory properties of high-dose omega-3 fatty acids and Vitamin D, and the initial encouraging results from case reports on the use of this supplementation in new-onset Type 1 Diabetes (T1D), support further testing of this combination strategy. This intervention appears to be well tolerated, affordable, and sufficiently safe to be further tested in randomized prospective trials to determine whether this combination therapy may be of assistance to halt progression of autoimmunity and/or preserve residual beta-cell function in subjects with new onset and established T1D of up to 10 years duration. In addition, the 1st PreDiRe T1D conference (Preventing Disease and its Recurrence in Type 1 Diabetes - see Editorial in this issue) was organized to discuss initial results and possible alternative/complementary strategies, for collaborative international expansion of these trials, to include strategies for disease prevention. Our POSEIDON clinical trial will test the use of high dose vitamin D3 and highly purified Omega-3 fatty acids in new onset and established T1D. The draft of the study protocol, in addition to the informed consent and assent, is now shared open access to facilitate its international implementation by interested physicians and centers that would like to further test this approach through clinical trials.</p>","PeriodicalId":87230,"journal":{"name":"CellR4-- repair, replacement, regeneration, & reprogramming","volume":"6 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2018-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8025938/pdf/nihms-1056744.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25590187","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Purified Human Pancreatic Islets, CIT Transplant Wash Media with Lisofylline.","authors":"","doi":"","DOIUrl":"","url":null,"abstract":"","PeriodicalId":87230,"journal":{"name":"CellR4-- repair, replacement, regeneration, & reprogramming","volume":"5 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2017-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6319638/pdf/nihms-997673.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36881102","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Purified Human Pancreatic Islets, CIT Culture Media with Lisofylline or Exenatide.","authors":"J Ansite,&nbsp;A N Balamurugan,&nbsp;B Barbaro,&nbsp;J Battle,&nbsp;D Brandhorst,&nbsp;J Cano,&nbsp;X Chen,&nbsp;S Deng,&nbsp;D Feddersen,&nbsp;A Friberg,&nbsp;T Gilmore,&nbsp;J S Goldstein,&nbsp;E Holbrook,&nbsp;A Khan,&nbsp;T Kin,&nbsp;J Lei,&nbsp;E Linetsky,&nbsp;C Liu,&nbsp;X Luo,&nbsp;K McElvaney,&nbsp;Z Min,&nbsp;J Moreno,&nbsp;D O'Gorman,&nbsp;K K Papas,&nbsp;G Putz,&nbsp;C Ricordi,&nbsp;G Szot,&nbsp;T Templeton,&nbsp;L Wang,&nbsp;J J Wilhelm,&nbsp;J Willits,&nbsp;T Wilson,&nbsp;X Zhang,&nbsp;J Avila,&nbsp;B Begley,&nbsp;J Cano,&nbsp;S Carpentier,&nbsp;E Holbrook,&nbsp;J Hutchinson,&nbsp;C P Larsen,&nbsp;J Moreno,&nbsp;M Sears,&nbsp;N A Turgeon,&nbsp;D Webster,&nbsp;S Deng,&nbsp;J Lei,&nbsp;J F Markmann,&nbsp;N D Bridges,&nbsp;C W Czarniecki,&nbsp;J S Goldstein,&nbsp;G Putz,&nbsp;T Templeton,&nbsp;T Wilson,&nbsp;T L Eggerman,&nbsp;P Al-Saden,&nbsp;J Battle,&nbsp;X Chen,&nbsp;A Hecyk,&nbsp;H Kissler,&nbsp;X Luo,&nbsp;M Molitch,&nbsp;N Monson,&nbsp;E Stuart,&nbsp;A Wallia,&nbsp;L Wang,&nbsp;S Wang,&nbsp;X Zhang,&nbsp;D Bigam,&nbsp;P Campbell,&nbsp;P Dinyari,&nbsp;T Kin,&nbsp;N Kneteman,&nbsp;J Lyon,&nbsp;A Malcolm,&nbsp;D O'Gorman,&nbsp;C Onderka,&nbsp;R Owen,&nbsp;R Pawlick,&nbsp;B Richer,&nbsp;S Rosichuk,&nbsp;D Sarman,&nbsp;A Schroeder,&nbsp;P A Senior,&nbsp;A M J Shapiro,&nbsp;L Toth,&nbsp;V Toth,&nbsp;W Zhai,&nbsp;K Johnson,&nbsp;J McElroy,&nbsp;A M Posselt,&nbsp;M Ramos,&nbsp;T Rojas,&nbsp;P G Stock,&nbsp;G Szot,&nbsp;B Barbaro,&nbsp;J Martellotto,&nbsp;J Oberholzer,&nbsp;M Qi,&nbsp;Y Wang,&nbsp;L Bayman,&nbsp;K Chaloner,&nbsp;W Clarke,&nbsp;J S Dillon,&nbsp;C Diltz,&nbsp;G C Doelle,&nbsp;D Ecklund,&nbsp;D Feddersen,&nbsp;E Foster,&nbsp;L G Hunsicker,&nbsp;C Jasperson,&nbsp;D-E Lafontant,&nbsp;K McElvaney,&nbsp;T Neill-Hudson,&nbsp;D Nollen,&nbsp;J Qidwai,&nbsp;H Riss,&nbsp;T Schwieger,&nbsp;J Willits,&nbsp;J Yankey,&nbsp;R Alejandro,&nbsp;A C Corrales,&nbsp;R Faradji,&nbsp;T Froud,&nbsp;A A Garcia,&nbsp;E Herrada,&nbsp;H Ichii,&nbsp;L Inverardi,&nbsp;N Kenyon,&nbsp;A Khan,&nbsp;E Linetsky,&nbsp;J Montelongo,&nbsp;E Peixoto,&nbsp;K Peterson,&nbsp;C Ricordi,&nbsp;J Szust,&nbsp;X Wang,&nbsp;M H Abdulla,&nbsp;J Ansite,&nbsp;A N Balamurugan,&nbsp;M D Bellin,&nbsp;M Brandenburg,&nbsp;T Gilmore,&nbsp;J V Harmon,&nbsp;B J Hering,&nbsp;R Kandaswamy,&nbsp;G Loganathan,&nbsp;K Mueller,&nbsp;K K Papas,&nbsp;J Pedersen,&nbsp;J J Wilhelm,&nbsp;J Witson,&nbsp;C Dalton-Bakes,&nbsp;H Fu,&nbsp;M Kamoun,&nbsp;J Kearns,&nbsp;Y Li,&nbsp;C Liu,&nbsp;E Luning-Prak,&nbsp;Y Luo,&nbsp;E Markmann,&nbsp;Z Min,&nbsp;A Naji,&nbsp;M Palanjian,&nbsp;M Rickels,&nbsp;R Shlansky-Goldberg,&nbsp;K Vivek,&nbsp;A S Ziaie,&nbsp;L Fernandez,&nbsp;D B Kaufman,&nbsp;L Zitur,&nbsp;D Brandhorst,&nbsp;A Friberg,&nbsp;O Korsgren","doi":"","DOIUrl":"","url":null,"abstract":"","PeriodicalId":87230,"journal":{"name":"CellR4-- repair, replacement, regeneration, & reprogramming","volume":"5 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2017-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6319648/pdf/nihms-997662.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36881106","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Purified Human Pancreatic Islets, CIT Culture Media with Lisofylline.","authors":"","doi":"","DOIUrl":"","url":null,"abstract":"","PeriodicalId":87230,"journal":{"name":"CellR4-- repair, replacement, regeneration, & reprogramming","volume":"5 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2017-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6319656/pdf/nihms-997667.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36881108","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Purified Human Pancreatic Islets with Lisofylline, Interim Certificate of Analysis (Product Code PHPI-L-01).","authors":"","doi":"","DOIUrl":"","url":null,"abstract":"","PeriodicalId":87230,"journal":{"name":"CellR4-- repair, replacement, regeneration, & reprogramming","volume":"5 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2017-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6319624/pdf/nihms-997531.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36881096","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}