Skin transplants could treat diabetes and obesity – Futurity: Research News
Posted: August 10, 2017 at 5:43 pm
Skin transplantation could be an effective way to deliver gene therapy to treat type 2 diabetes and obesity, new research in mice suggests.
The technique could enable a wide range of gene-based therapies to treat many human diseases.
We think this can provide a long-term safe option for the treatment of many diseases
We resolved some technical hurdles and designed a mouse-to-mouse skin transplantation model in animals with intact immune systems, says study author Xiaoyang Wu, assistant professor in the cancer research department at the University of Chicago.
We think this platform has the potential to lead to safe and durable gene therapy in mice and, we hope, in humans, using selected and modified cells from skin.
Beginning in the 1970s, physicians learned how to harvest skin stem cells from a patient with extensive burn wounds, grow them in the laboratory, then apply the lab-grown tissue to close and protect a patients wounds. This approach is now standard. However, the application of skin transplants is better developed in humans than in mice.
The mouse system is less mature, Wu says. It took us a few years to optimize our 3D skin organoid culture system.
This study is the first to show that an engineered skin graft can survive long term in wild-type mice with intact immune systems.
We have a better than 80 percent success rate with skin transplantation, Wu says. This is exciting for us.
The researchers focused on diabetes because it is a common non-skin disease that can be treated by the strategic delivery of specific proteins.
They inserted the gene for glucagon-like peptide 1 (GLP1), a hormone that stimulates the pancreas to secrete insulin. This extra insulin removes excessive glucose from the bloodstream, preventing the complications of diabetes. GLP1 can also delay gastric emptying and reduce appetite.
Using CRISPR, a tool for precise genetic engineering, they modified the GLP1 gene. They inserted one mutation, designed to extend the hormones half-life in the blood stream, and fused the modified gene to an antibody fragment so that it would circulate in the blood stream longer. They also attached an inducible promoter, which enabled them to turn on the gene to make more GLP1, as needed, by exposing it to the antibiotic doxycycline. Then they inserted the gene into skin cells and grew those cells in culture.
When these cultured cells were exposed to an air/liquid interface in the laboratory, they stratified, generating what the authors referred to as a multi-layered, skin-like organoid.
Next, they grafted this lab-grown gene-altered skin onto mice with intact immune systems. There was no significant rejection of the transplanted skin grafts.
When the mice ate food containing minute amounts of doxycycline, they released dose-dependent levels of GLP1 into the blood. This promptly increased blood-insulin levels and reduced blood-glucose levels.
When the researchers fed normal or gene-altered mice a high-fat diet, both groups rapidly gained weight. They became obese. When normal and gene-altered mice got the high-fat diet along with varying levels of doxycycline, to induce GLP1 release, the normal mice grew fat and mice expressing GLP1 showed less weight gain.
Expression of GLP1 also lowered glucose levels and reduced insulin resistance.
Together, our data strongly suggest that cutaneous gene therapy with inducible expression of GLP1 can be used for the treatment and prevention of diet-induced obesity and pathologies, the authors write.
When they transplanted gene-altered human cells to mice with a limited immune system, they saw the same effect. These results, the authors wrote, suggest that cutaneous gene therapy for GLP1 secretion could be practical and clinically relevant.
This approach, combining precise genome editing in vitro with effective application of engineered cells in vivo, could provide significant benefits for the treatment of many human diseases, the authors note.
We think this can provide a long-term safe option for the treatment of many diseases, Wu says. It could be used to deliver therapeutic proteins, replacing missing proteins for people with a genetic defect, such as hemophilia. Or it could function as a metabolic sink, removing various toxins.
Skin progenitor cells have several unique advantages that are a perfect fit for gene therapy. Human skin is the largest and most accessible organ in the body. It is easy to monitor. Transplanted skin can be quickly removed if necessary. Skins cells rapidly proliferate in culture and can be easily transplanted. The procedure is safe, minimally invasive, and inexpensive.
There is also a need. More than 100 million US adults have either diabetes (30.3 million) or prediabetes (84.1 million), according the Centers for Disease Control and Prevention. More than two out of three adults are overweight. More than one out of three are considered obese.
Additional authors of the study are from the University of Chicago and the University of Illinois at Chicago. The National Institutes of Health, the American Cancer Society, and the V Foundation funded the study.
Source: University of Chicago
Follow this link:
Skin transplants could treat diabetes and obesity - Futurity: Research News
- This Pitt program is leveling up the gene therapy workforce in Pittsburgh - University of Pittsburgh - March 12th, 2024
- Opinion | Banning Gain-of-Function Research Would Do Far More Harm Than Good - Medpage Today - March 12th, 2024
- The Biggest Challenges Facing Broader Adoption of Novel Cell and Gene Therapies - Pharmaceutical Executive - March 12th, 2024
- Research and development areas of focus - Bristol Myers Squibb - March 4th, 2024
- Overcoming the challenges of gene therapy - Drug Discovery News - March 4th, 2024
- Breakthrough Gene Therapy for LHON: Vision Restoration in Focus at Medical Conference - BNN Breaking - March 4th, 2024
- Revolutionizing Healthcare: Global Gene Therapy Market Surges to US$ 3.44 Billion by 2022 As Revealed In Ne... - WhaTech - March 4th, 2024
- Looking to the future of clinical trials: Gene therapy, precision medicine, and the ongoing quest for rare disease solutions - pharmaphorum - March 4th, 2024
- Complement system response to AAV vector gene therapy - EurekAlert - February 23rd, 2024
- 10 gene therapy companies you should know about - Labiotech.eu - February 23rd, 2024
- Fetal surgery expert Tippi MacKenzie shifts to in-utero gene therapy - STAT - February 23rd, 2024
- Rejuvenate Bio Announces Gene Therapy-Mediated Partial Reprogramming Extends Lifespan and Reverses Age ... - Business Wire - February 23rd, 2024
- Combatting HIV using gene therapy - Drug Target Review - February 23rd, 2024
- Expanding the Horizons of Cell and Gene Therapy - RegMedNet - February 23rd, 2024
- Global Advanced Therapy Medicinal Products CDMO Market Analysis Report 2024-2030: : Increasing Investment and ... - Yahoo Finance UK - February 23rd, 2024
- 2023 gene therapy research STAR Grant winners announced - Pacific Biosciences - February 13th, 2024
- Lifetime Cost of DEB Gene Therapy Could Be $15-$22 Million - Medscape - February 13th, 2024
- 5 Things Youll Learn in Frederick Community Colleges New Cell and Gene Therapy Essentials Certificate ... - BioBuzz - February 13th, 2024
- Regenxbios investigational gene therapy shows promise in Hunter syndrome - PMLiVE - February 13th, 2024
- Topical Gene Therapy Effective in Treating Debilitating Eye Condition - InventUM - University of Miami - February 13th, 2024
- Bayer Says First Patient Randomized In Askbio Phase II Gene Therapy Trial - XM - February 13th, 2024
- Top 3 grants in regenerative medicine: January 2024 - RegMedNet - February 13th, 2024
- Realistic Expectations, Limitations With Gene Therapy for Parkinson Disease: Ignacio Mata, PhD - Neurology Live - February 5th, 2024
- Gene-Based Therapy May Slow Development of Life-Threatening Heart Condition - NYU Langone Health - February 5th, 2024
- After Promising Early Efficacy, Eli Lilly Eager to Study Hearing Loss Gene Therapy in More Children - precisionmedicineonline.com - February 5th, 2024
- Gene therapy hailed as medical magic wand for hereditary swelling disorder - The Guardian - February 5th, 2024
- France Approves Gene Therapy Clinical Trial to Treat Hearing Loss in Children - The Hearing Review - February 5th, 2024
- Early Trial Success for Inherited Deafness Gene-Therapy - RegMedNet - February 5th, 2024
- Can gene therapy be the key to restoring hearing loss? - Labiotech.eu - February 5th, 2024
- Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum ... - Business Wire - February 5th, 2024
- FDA inspection clears Abeona's gene therapy BLA By Investing.com - Investing.com - February 5th, 2024
- Hearing Restored in Deaf Children in Gene Therapy Clinical Trial - Securities.io - February 5th, 2024
- Experimental gene therapy allows kids with inherited deafness to hear - The Associated Press - January 28th, 2024
- Children with genetic deafness have hearing restored with gene therapy: Study - ABC News - January 28th, 2024
- Positive Phase 1/2 Clinical Trial Data for an Investigational Gene Therapy for Genetic Hearing Loss to be Presented at ... - Investors | Eli Lilly and... - January 28th, 2024
- UChicago Medicine among the first in the country to offer newly approved sickle cell gene therapies - UChicago Medicine - January 28th, 2024
- Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time - The New York Times - January 28th, 2024
- Gene therapy restores hearing in children with hereditary deafness - EurekAlert - January 28th, 2024
- Experimental gene therapy helps treat kids with inherited hearing loss - CBS News - January 28th, 2024
- Children's Hospital of Philadelphia Performs First in U.S. Gene Therapy Procedure to Treat Genetic Hearing Loss - PR Newswire - January 28th, 2024
- Gene Therapy Brings Hearing to Kids With Congenital Deafness - U.S. News & World Report - January 28th, 2024
- Gene Therapy Restores Hearing in Small Trial for Kids With Hereditary Deafness - Technology Networks - January 28th, 2024
- Cell Therapies on Ice: Seven Trends in Cryopreservation To Enable Cell and Gene Therapy Research - Technology Networks - January 28th, 2024
- Gene Therapy Brings Hearing to Kids With Congenital Deafness - HealthDay - January 28th, 2024
- Gene therapies restore hearing in several kids with inherited deafness - Livescience.com - January 28th, 2024
- 11-Year-Old Boy Hears For First Time Ever Thanks To Gene Therapy Breakthrough - IFLScience - January 28th, 2024
- Innovative Gene Therapy Restores Hearing In Deaf Child, Offering Potential Cure - Study Finds - January 28th, 2024
- Sound of Success, Gene Therapy Breakthrough Grants Hearing to Deaf Children in China-Harvard Study - Hoodline - January 28th, 2024
- Perspectives in FTD Research Webinar: Gene Therapy for FTD -- What Do I Need to Know? | AFTD - AFTD - January 20th, 2024
- UK leads the way in cell and gene therapy - Research Professional News - January 20th, 2024
- Charles River's (CRL) New Launch to Boost Gene Therapy Programs - Yahoo Finance - January 20th, 2024
- UK maintains status as an attractive destination for advanced therapy clinical trials - BioPharma-Reporter.com - January 20th, 2024
- Understanding the impact of AI on the advancement of gene therapy development - pharmaphorum - January 20th, 2024
- Gene therapy TSHA-102 delivered to 1st child in REVEAL trial - Rett Syndrome News - January 20th, 2024
- AAV Vectors in Gene Therapy Market is Predicted to Observe Skyrocketed Growth During the Study Period (2019-2032 ... - PR Newswire - January 20th, 2024
- Family pins hopes on gene therapy - The Recorder and Times - January 20th, 2024
- Arbor, 4DMT partner on new gene therapies in ALS, other diseases - ALS News Today - January 11th, 2024
- The year of gene therapy? FDA's Marks calls for solutions to regulatory, manufacturing challenges - BioWorld Online - January 11th, 2024
- Gene Therapy Approvals Expected to Ramp Up in 2024 Amid Manufacturing, Cost Challenges - BioSpace - January 11th, 2024
- Turning On Gene Therapy with the pA Regulator - RegMedNet - January 11th, 2024
- Revolutionizing Healthcare: Gene Therapy Market Projected to Reach USD 46.5 billion by 2030 at a 22. - PharmiWeb.com - January 11th, 2024
- Regeneron CEO Reveals the Game-Changing Potential of Gene Therapy in Biotech! - BioTecNika - January 11th, 2024
- Bayer's AskBio checks off another early win for gene therapy platform with Parkinson's safety data - FierceBiotech - January 11th, 2024
- Are Rare Brain Diseases the Next Commercial Frontier for Gene Therapy? - MedCity News - January 3rd, 2024
- Novartis, Voyager Ink Up-to-$1.3B Expansion of Neuro Gene Therapy Collaboration - Genetic Engineering & Biotechnology News - January 3rd, 2024
- Novel switch turns genes on/off on cue, a promising step toward safer gene therapy - Phys.org - January 3rd, 2024
- Which Therapeutic Areas Experts Think Will Gain Traction In 2024 - Clinical Leader - January 3rd, 2024
- Voyager Therapeutics and Novartis Announce Major Licensing Deal in Gene Therapy Field - Medriva - January 3rd, 2024
- We Are About to Enter the Golden Age of Gene Therapy - Inverse - January 3rd, 2024
- Revolutionizing Gene Therapy: Baylor Researchers Develop RNA-Based Switch for Therapeutic Protein Control - BNN Breaking - January 3rd, 2024
- Casgevy and Lyfgenia: Two Gene Therapies Approved for Sickle Cell Disease - Yale Medicine - December 25th, 2023
- Top story: Family connects with researchers behind Canavan gene therapy - UMass Medical School - December 25th, 2023
- Excision Bio Seeks to Suppress HIV Replication With CRISPR Gene Therapy - AJMC.com Managed Markets Network - December 25th, 2023
- Hemgenix Gene Therapy Shows Long-Term Efficacy, Safety in Hemophilia B Patients - Managed Healthcare Executive - December 25th, 2023
- The missing piece of the puzzle - EUobserver - December 25th, 2023
- Brace yourselves for these trends in clinical research in 2024 - Labiotech.eu - December 25th, 2023
- RegMedNet's 2023 remix: top content of the year - RegMedNet - December 25th, 2023
- BU Researchers Helped Develop the First FDA-Approved Gene Therapies to Treat Sickle Cell Disease - Boston University - December 16th, 2023
- Research discovers that mice possess a natural gene therapy system - Phys.org - December 16th, 2023
- Cell- and gene-therapy hub bound for Lake Success; New York to invest up to $150 million - Newsday - December 16th, 2023