Contact Information
Available for logged-in reporters only
Newswise Geneticists from Ohio, California and Japan joined forces in a quest to correct a faulty chromosome through cellular reprogramming. Their study, published online today in Nature, used stem cells to correct a defective ring chromosome with a normal chromosome. Such therapy has the promise to correct chromosome abnormalities that give rise to birth defects, mental disabilities and growth limitations.
In the future, it may be possible to use this approach to take cells from a patient that has a defective chromosome with multiple missing or duplicated genes and rescue those cells by removing the defective chromosome and replacing it with a normal chromosome, said senior author Anthony Wynshaw-Boris, MD, PhD, James H. Jewell MD '34 Professor of Genetics and chair of Case Western Reserve School of Medicine Department of Genetics and Genome Sciences and University Hospitals Case Medical Center.
Wynshaw-Boris led this research while a professor in pediatrics, the Institute for Human Genetics and the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UC, San Francisco (UCSF) before joining the faculty at Case Western Reserve in June 2013.
Individuals with ring chromosomes may display a variety of birth defects, but nearly all persons with ring chromosomes at least display short stature due to problems with cell division. A normal chromosome is linear, with its ends protected, but with ring chromosomes, the two ends of the chromosome fuse together, forming a circle. This fusion can be associated with large terminal deletions, a process where portions of the chromosome or DNA sequences are missing. These deletions can result in disabling genetic disorders if the genes in the deletion are necessary for normal cellular functions.
The prospect for effective counter measures has evaded scientistsuntil now. The international research team discovered the potential for substituting the malfunctioning ring chromosome with an appropriately functioning one during reprogramming of patient cells into induced pluripotent stem cells (iPSCs). iPSC reprogramming is a technique that was developed by Shinya Yamanaka, MD, PhD, a co-corresponding author on the Nature paper. Yamanaka is a senior investigator at the UCSF-affiliated Gladstone Institutes, a professor of anatomy at UCSF, and the director of the Center for iPS Cell Research and Application (CiRA) at the Institute for Integrated Cell-Material Sciences (iCeMS) in Kyoto University. He won the Nobel Prize in Medicine in 2012 for developing the reprogramming technique.
Marina Bershteyn, PhD, a postdoctoral fellow in the Wynshaw-Boris lab at UCSF, along with Yohei Hayashi, PhD, a postdoctoral fellow in the Yamanaka lab at the Gladstone Institutes, reprogrammed skin cells from three patients with abnormal brain development due to a rare disorder called Miller Dieker Syndrome, which results from large terminal deletions in one arm of chromosome 17. One patient had a ring chromosome 17 with the deletion and the other two patients had large terminal deletions in one of their chromosome 17, but not a ring. Additionally, each of these patients had one normal chromosome 17.
The researchers observed that, after reprogramming, the ring chromosome 17 that had the deletion vanished entirely and was replaced by a duplicated copy of the normal chromosome 17. However, the terminal deletions in the other two patients remained after reprogramming. To make sure this phenomenon was not unique to ring chromosome 17, they reprogrammed cells from two different patients that each had ring chromosomes 13. These reprogrammed cells also lost the ring chromosome, and contained a duplicated copy of the normal chromosome 13.
It appears that ring chromosomes are lost during rapid and continuous cell divisions during reprogramming, said Yamanaka. The duplication of the normal chromosome then corrects for that lost chromosome.
See original here:
Nature Study Discovers Chromosome Therapy to Correct a Severe Chromosome Defect
- Immunotherapy Inches Forward in Development of Myeloid Malignancies - OncLive - January 14th, 2021
- Cytovia Therapeutics Partners with National Cancer Institute to Develop Novel Gene-Edited, iPSC-Derived GPC3 CAR NK Cells for the Treatment of Solid... - January 14th, 2021
- Kaleido Biosciences Announces Positive Interim Results of Controlled Study of KB109 in Patients with Mild-to-Moderate COVID-19 - BioSpace - January 14th, 2021
- Doctors Make Medical Breakthrough In Treating Severe Cases Of COVID - CBS San Francisco - January 9th, 2021
- Mana joins the hectic fight against solid tumors with an 'off-the-shelf' candidate angling for an IND this year - Endpoints News - January 9th, 2021
- Versiti Blood Centers and Noodles & Company Serve Up Thanks to Blood Donors - PRNewswire - December 31st, 2020
- Global CAR-T Pipeline Insight Report 2020: Overview, Landscape, Therapeutic Assessment, Current Treatment Scenario and Emerging Therapies -... - December 31st, 2020
- 2020 health care year in review - Crain's Cleveland Business - December 31st, 2020
- The Top 10 FDA Oncology Drug Approvals of 2020 - Curetoday.com - December 31st, 2020
- Gut microbiota: How does it interact with the brain? - Medical News Today - December 31st, 2020
- Numerous Indian American STEM Researchers Named Fellows of American Association for the Advancement of Sciences - India West - December 31st, 2020
- Four years after devastating spinal injury, former St. Paul's football player reunites with caregivers - NOLA.com - December 24th, 2020
- New Combination Therapy Tested By Children's May Offer Hope For Leukemia Patients - WVXU - December 24th, 2020
- Real-time observation helpful in Stem cell for vascular diseases: Study - Hindustan Times - December 21st, 2020
- NurOwn May Be Given to Early ALS Patients in US Who Finished Phase... - ALS News Today - December 21st, 2020
- Follow the Money: Spatial Omics, CAR-NK Cells, AI-Powered Biology - Bio-IT World - December 21st, 2020
- Explained: Process of transporting stem cell from donor to patient for a successful transplant - Firstpost - December 18th, 2020
- Research That Saves Lives: Four COVID-19 Therapies Being Tested at UVA - University of Virginia - December 18th, 2020
- Even if You've Had COVID-19 You Still Need the Vaccine - Healthline - December 18th, 2020
- What Patients With Cancer, Survivors Need to Know About the Emergency Use Authorization of COVID-19 Vaccine - Curetoday.com - December 16th, 2020
- 2020 at the U: The year in review - University of Miami - December 16th, 2020
- Five Mobile County hospitals to get Pfizer vaccine this week - AL.com - December 16th, 2020
- Donor Stem Cell Transplant Improves Survival in Older Patients with Myelodysplastic Syndrome - Cancer Health Treatment News - December 10th, 2020
- Positive Phase 2 Proof-of-Concept Data for Viralym-M and Burden of Disease Data Presented in Oral Presentations at the 62nd American Society of... - December 10th, 2020
- Dr. Kansagra: Quadruplet Therapy for Newly Diagnosed Multiple Myeloma and Combination CAR T-Cell Opportunities - DocWire News - December 10th, 2020
- Silicon Therapeutics Announces Members of Scientific Advisory Board - Business Wire - December 10th, 2020
- Israeli Neurogenesis' NG-01 slows progressive MS by up to 90% in phase II study - BioWorld Online - December 10th, 2020
- ADC Therapeutics Announces Updated Clinical Data on Lead Antibody Drug Conjugate Programs Loncastuximab Tesirine (Lonca) and Camidanlumab Tesirine... - December 10th, 2020
- Magenta Therapeutics Announces Commencement of First Phase 2 Clinical Trial of MGTA-145 for Stem Cell Mobilization, Oral Presentation of MGTA-145... - December 8th, 2020
- Precigen Presents New Data Supporting the Safety, Clinical Activity, Expansion and Persistence of PRGN-3006 UltraCAR-T at the 62nd ASH Annual Meeting... - December 8th, 2020
- ALLO-715, Off-the-Shelf CAR T-Cell Therapy, Produces Early Promise in Multiple Myeloma - Cancer Network - December 8th, 2020
- Rocket Pharmaceuticals Presents Positive Clinical Data from its Fanconi Anemia and Leukocyte Adhesion Deficiency-I Programs at the 62nd American... - December 8th, 2020
- Negrin Shines Light on the Orca-T Story in GVHD - OncLive - December 8th, 2020
- Preliminary Results from NexImmune's Phase 1/2 Trial of NEXI-001 in AML Presented at 62nd ASH Annual Meeting and Exposition - GlobeNewswire - December 8th, 2020
- BeiGene Announces the Approval in China of BLINCYTO (Blinatumomab) for Injection for Adult Patients with Relapsed or Refractory B-Cell Precursor Acute... - December 8th, 2020
- Sutro Biopharma Presents Data from Ongoing Phase 1 Dose-Escalation Study for STRO-001 for the Treatment of B-cell Non-Hodgkin Lymphoma at the 62nd... - December 8th, 2020
- Data from the ANDROMEDA Study Show Hematologic Response for DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) in Newly Diagnosed Light Chain (AL)... - December 8th, 2020
- US FDA Approves Naxitamab for the Treatment of Neuroblastoma - OncoZine - November 29th, 2020
- Coronavirus treatments and vaccines. Here are the latest developments - San Francisco Chronicle - November 29th, 2020
- Dr Apar Kishor Ganti Outlines the Effectiveness of Lurbinectedin and Benefits Over Competition - AJMC.com Managed Markets Network - November 26th, 2020
- Commonly used antibiotic shows promise for combating Zika infections - National Institutes of Health - November 26th, 2020
- A real life Superman celebrates 5 years of survival from one of the deadliest cancers - Newswise - November 26th, 2020
- Global Cell Harvesting Market to Reach US$381,4 Million by the Year 2027 - Salamanca Press - November 26th, 2020
- Tanya Siddiqi, MD, Discusses the Promise of Reduced Toxicity With Liso-Cel - AJMC.com Managed Markets Network - November 26th, 2020
- Tetracycline-based Antibiotics Show Promise for Combating Zika Infections - Global Biodefense - November 26th, 2020
- Study Suggests AYAs Treated for AML Have High Risk of Developing Long-Term Complications - Cancer Network - November 26th, 2020
- Mustang Bio Announces Positive Opinion from the European Medicines Agency on Orphan Drug Designation for Its Lentiviral Gene Therapy for the Treatment... - November 26th, 2020
- Study Finds Racial and Ethnic Disparities in Treatment of AL Amyloidosis - DocWire News - November 22nd, 2020
- New Virtual Reality Tool, Domestic Cats are SARS-CoV-2 Carriers, Combination Therapy Advances: COVID-19 Updates - Bio-IT World - November 22nd, 2020
- SEE | Smoking worsens Covid-19 infection in the airways, new study reveals - Health24 - November 22nd, 2020
- Could Cell Therapy Be The Answer To COVID-19? - CBS Boston - November 3rd, 2020
- CA Prop 14 Explained: What To Know Before You Vote Election Day - Los Angeles, CA Patch - November 3rd, 2020
- GMP Cell Banking Services Market Predicted to Accelerate the Growth by 2019-2029 - TechnoWeekly - November 3rd, 2020
- Making Progress Against Relapsed/ Refractory DLBCL Without CAR T - OncLive - November 3rd, 2020
- Orchard Therapeutics to Host Virtual R&D Investor Event on Friday, November 13, 2020 - GlobeNewswire - November 3rd, 2020
- Editorial Article: Combating cancer: The incubation technology accelerating CAR-T cell therapy development - SelectScience - October 31st, 2020
- Richters Transformation: When a Slow-Growing Cancer Turns Aggressive - SurvivorNet - October 31st, 2020
- Detroit Pistons, Make-A-Wish put a smile on the face of Commerce Township boy - The Oakland Press - October 31st, 2020
- NexImmune Establishes Research Initiative with City of Hope to Focus on Novel Immunotherapeutic Approaches to Acute Myeloid Leukemia - Yahoo Finance - October 31st, 2020
- HBS and Harvard's Department of Stem Cell and Regenerative Biology Receive $25 Million Gift from The Chris and Carrie Shumway Foundation to Support... - October 23rd, 2020
- Harnessing regeneration of retinal tissues: An option almost within reach - Ophthalmology Times - October 23rd, 2020
- Fate Therapeutics' and Celyad's CAR therapies in oncology offer potential - pharmaceutical-technology.com - October 23rd, 2020
- World Wide Stem Cell Treatment Centers Provides Exceptional Regenerative Treatment Options To Those in Need - GlobeNewswire - October 16th, 2020
- Proposition 14: Stem cell research bonds City Times - City Times - October 16th, 2020
- BrainStorm Announces Financial Results for the Third Quarter of 2020 and Provides a Corporate Update - PRNewswire - October 16th, 2020
- How a UCSF team is giving Cronutt the sea lion a second chance with neuroscience - University of California - October 16th, 2020
- Caliber Pain is Featured as a 2020 Top NYC Patient Rated Pain Management Practice by Find Local Doctors - PR Web - October 10th, 2020
- Report of the president: Appointments and promotions | Stanford News - Stanford University News - October 10th, 2020
- UTSW Scientist: 'We Need More Women Thinking Creatively in the Lab and Pushing Science Forward' - dallasinnovates.com - October 10th, 2020
- Here's what is known about Trump's COVID-19 treatment - Science Magazine - October 4th, 2020
- Election Guide: Here's What You Need to Know About Proposition 14 - NBC Bay Area - October 4th, 2020
- ASX up 2.3%, banks and energy outperform - The Sydney Morning Herald - October 4th, 2020
- Autologous Stem Cell and Non-Stem Cell Based Therapies MARKET 2020 |INDUSTRY ANALYSIS, SIZE, SHARE, TRENDS, MARKET DEMAND, GROWTH, OPPORTUNITIES AND... - September 25th, 2020
- Finding The Achilles Heel of A Killer Parasite - Newswise - September 25th, 2020
- SHE Initiative Shines Light on Gender Disparities in Oncology Workforce - Targeted Oncology - September 25th, 2020
- Actinium Pharmaceuticals Successfully Completes First Dosing Cohort in the Phase 1 Study of Actimab-A and Venetoclax Combination Therapy in... - September 23rd, 2020
- Advances in the Treatment of Mantle Cell Lymphoma are Greatly Improving the Long-Term Outlook for Patients - Curetoday.com - September 23rd, 2020
- CytoDyn's Phase 2 Study of Leronlimab for Mild-to-Moderate COVID-19 Selected for Oral Presentation at the Special isirv-AVG Virtual Conference on... - September 23rd, 2020
- Opdivo (nivolumab) Demonstrated Superior Disease-Free Survival in Patients with Resected Esophageal or Gastroesophageal Junction Cancer Compared to... - September 23rd, 2020
- Coronavirus Researchers Discover How COVID-19 May Trigger Fatal Levels of Lung Inflammation - SciTechDaily - September 21st, 2020