Research Progress, December 2021

Research Progress, December 2021
December 3, 2021 Dear All, I hope you and your family are doing well. We live in a time of tumult. Many are wary of all the negativity, but I am incredibly positive and energetic and committed to making a difference. Our lab is 100% functional, and we are making steady progress. Here is a […]

Updates – an upcoming trial

Updates – an upcoming trial
May 30, 2021 Dear All, Hello everyone. I pray you’re in great spirits. Everyone in my research team has been vaccinated for COVID-19, and we are working very hard to develop novel treatments for Wolfram syndrome. Although our past has been harsh, our future looks brilliant now. I continue adhering to my three guiding principles: […]

A rare and amazing opportunity

As many of you are aware, Science Magazine featured my basic, translational, and clinical research on Wolfram syndrome. The article covers the history of Wolfram syndrome and its research. https://www.sciencemag.org/news/2021/02/race-treat-rare-fatal-syndrome-may-help-others-common-disorders-diabetes https://science.sciencemag.org/content/371/6530/663 This is a rare and amazing opportunity for us to raise awareness of Wolfram syndrome. Science initially interviewed me on endoplasmic reticulum stress (ER […]

Updates on Therapeutic Development

February 6, 2021 Dear Friends, I wish and pray that 2021 is a year of splendid health and big success for you. Despite all the uncertainty, troubles, and challenges on our planet, I have focused my mind and efforts on helping and saving our patients with Wolfram syndrome and WFS1-related disorders. I have been adhering […]

Therapeutic Development Pipelines and Timeline

Therapeutic Development Pipelines and Timeline
Dear Friends, First of all, I want to express my gratitude for everything you’ve helped me achieve here. Your kind words and encouragement keep me motivated and inspired. My three guiding principles are: Improve clinical care, Raise awareness, and Provide a cutting-edge treatment for Wolfram syndrome. Here is our progress: A Drug-Repurposing Clinical Trial Our […]

Continue working as one team

Dear Friends, Thank you for visiting our Wolfram Syndrome website. I hope you and your family are safe, strong, and healthy on a brighter future. I have no doubt better days are ahead, and we can rise about this crisis. Many of you have asked me if I need to assist with COVID-19 research or […]

My deepest and sincere gratitude to you

My deepest and sincere gratitude to you
Dear Friends, Today, I would like to show my gratitude to you. I have received hundreds of emails in the past few days regarding our recent advances in regenerative gene therapy for Wolfram Syndrome that could lead to breakthrough therapy not only for Wolfram Syndrome but also for retinal degeneration, diabetes, and neurodegenerative disorders. All […]

Toward personalized regenerative gene therapy for Wolfram syndrome

Toward personalized regenerative gene therapy for Wolfram syndrome
Dear Friends, I hope you and your family are safe and well during this COVID-19 pandemic. Three things are always on my mind: Improve clinical care, Raise awareness, and Provide a cutting-edge treatment for Wolfram syndrome. As I mentioned on January 1st, I am determined to make 2020 the game-changing year for us despite this […]

Rare Disease Day 2020

Rare Disease Day 2020
Dear Friends, Happy Rare Disease Day. Thank you for your emails and messages in the past few days. I appreciate you shared your feelings with me and encouraged me regarding our therapeutic development for Wolfram syndrome. As I mentioned in my blog on January 1, 2020, I am determined to make 2020 the game-changing year […]

Bringing regenerative therapies to patients

We have been developing drug-based therapies to delay/stop the progression of Wolfram Syndrome, and we have made significant progress. In parallel, we need to develop regenerative therapies to treat/replace damaged tissues. We have two pipelines for the regenerative therapies. 1. Stem cell-based therapies.This requires collaboration with surgeons and bioengineers. We are working on this.2. Regeneration […]

Goals for 2020: One Team

Goals for 2020: One Team
Dear Friends, Happy New Year. Thank you for your email, letters, and messages in the past few weeks. I appreciated your encouragement. I am determined to make 2020 the game-changing year for us. Three things are always on my mind: Improve clinical care, Raise awareness, and Provide a cutting-edge treatment for Wolfram syndrome. I have […]

Thank you for your support. Our progress in 2019.

Thank you for your support. Our progress in 2019.
Dear Friends, As the year 2019 is coming to an end, I would like to summarize our progress this year. Tomorrow, I would like to tell you my plan for 2020. We have been focusing our efforts on the development of regenerative and gene therapy. We successfully created the adeno-associated virus (AAV) that can deliver […]