Treat "cystic fibrosis" with nano machine!
Reached the funding target!
皆様
お世話になっております。ヨウです。
皆さまのご支援と応援、拡散のご協力をいただきまして、ありがとうございました！ 現状、目標金額の40万円を達成しました。達成率も104％と順調に推移しており、皆様のご支援につきまして、言葉もございません。
今回のクラウドファンディングを通して研究資金をいただけたことは非常に重要でしたが、自分自身の人生にとっても、非常に意義深いだと思います。最初クラウドファンディングのホームページを準備するとき、日本語の部分では、難しくて、ほかの人より、何倍の時間をかけて書き終わりました。このプロジェクトは人生のいい勉強になりました。私の研究への応援コメントにも非常に勇気づけられました。 CFに苦しんでいる患者さんと家族や、研究においては先生や研究員、学生さんやお医者さんと関わることが多いですが、他の多くの方にも支えられているということを再認識しました。 このコミュニケーションも私の研究に対するモチベーションもさらに向上しました。
残りの期間ではセカンドゴールとして５０万円までを目標として、シミュレーションに必要なソフトの購入費用や学会発表・論文投稿の費用に充てたいと考えております。ナノマシンが完成した後は、細胞実験、動物実験という流れで研究を進め、実用化を目指していきたいと考えています。
引き続きご支援・ご声援よろしくお願いします。
ヨウブンセイ
Comment from academist staff
Graduate student's challenge for radical treatment of rare diseases
Ryosuke Shibato
What is Gene Therapy?
The human body is comprised of over 60 trillion cells which are controlled by genes: subunits of DNA located in the cell nucleus that code for all of the information and programming of a cell. The DNA carries the genetic blueprint used to make all of the proteins that the cell requires. Every gene contains a particular set of instructions which codes for a specific protein. If the sequence of DNA is altered, abnormal proteins are produced. Gene therapy utilizes nucleic acids such as DNA or RNA as drugs to treat diseases by genes disruption, insertion or correction in order to modulate diseases caused by incorrect gene sequences. This strategy allows for treatment of diseases that cannot be treated with conventional small molecule drugs or biologics, such as protein or antibody therapy.
Enhanced gene insertion into cells by nanomedicine
Gene therapies using genes, aptamers, and antisense oligonucleotides have been showing promising results in the laboratory. However, full translation of such therapies into the clinic requires a formulation that can achieve efficient gene modification with high safety and minimal side effects. Unfortunately, nucleic acids are both highly inefficient and extremely immunogenic when administered alone, which significantly limits therapeutic applications. The human body has evolved to be highly resistant to gene modification, as this is the primary method of viral replication. Thus, direct injection of genetic material into the blood stream has nearly no effect, as the genes will be rapidly destroyed by nucleases or circulating immune cells. Moreover, due to the small size of the nucleic acids, they are easily excreted as urine from the kidneys, which in turn means that nucleic acid medicines must be delivered to the cells directly to evade kidney excretion. Therefore, the nanomedicine was developed; by covering the nucleic acid drug with a polymer, the size of the drug is increased, so the nucleic acid drug can be carried close to the target cells without being excreted as urine from the kidney.
Enhancing nanocarrier stability and efficiency
Although our current polymeric nanocarriers have enhanced gene delivery properties, after nanomedicines were administered in vivo, there are many macromolecules present in the body which may destroy nanomedicine, they may be broken. Furthermore, when the unbroken nanomedicines reach near the target cell, it can easily be taken up by an acidic container endosome before playing its role and can be digested by a lysosome. Thus, to overcome the bottlenecks of nucleic acid therapies, nanomedicines should be designed to evade recognition as a foreign body by living organisms, and also avoid being digested by an endosome and a lysosome.
Predicting stability and efficiency by simulation
To overcome the bottlenecks of nucleic acid therapies, we are currently introducing functional amino acids into our polymeric nanocarrier to enhance function and stability, including the ability to escape cellular destruction in the endosome, as well as reversibly crosslinking the nanocarrier to enhance stability in circulation while allowing for a stimuli responsive release of the gene into the cell. As amino acids are the building blocks of proteins, the addition of amino acids into our nanocarrier is not expected to have any increase in toxicity or adverse effects. As there are 100 amino acids to choose from, some amino acids have stronger interaction with nucleic acids and some of them have better endosome escape stability, so molecular dynamic simulations are being carried out to provide an exhaustive exploration at the atomic level.
Why we need your support
We would like to apply our gene therapy to treat the genetic disease cystic fibrosis (CF). Cystic fibrosis is a genetic disease which occurs in roughly 1 in 600,000 Japanese citizens. There are no effective drugs for treatment of CF, and it is classified as a rare disease by the Ministry of Health, Labor, and Welfare of Japan. As the exact cause of CF is known to be a mutation of the CFTR gene, we believe that genetic therapy to suppress the expression of the mutant gene can be an effective treatment that could enhance the quality of life for all CF patients. Our novel polymeric nanocarrier for genetic material is a promising design strategy for treating CF, and we hope that you can join us in our goal to treat CF.
Thank you for your support.
Profile
楊文倩
Project timeline
| Date | Plans |
|---|---|
| June 2019 | Challenge to crowdfunding |
| August 2019 | Start experiment |
| December 2019 | Patent application |
| March 2020 | Article writing |
| 2020年5月 | 学会発表＠高分子学会 |
| 2021年5月 | 学会発表＠Annual meeting of Controlled Release Society |
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academist Prize 2nd adopted
