7 Gene Therapy Examples & Applications in Healthcare

Gene therapy is revolutionizing ヘルスケア and genomic medicine. The year 2017 demonstrated 遺伝子治療 癌をはじめとする致命的な疾患の治療につながる可能性のある事例やアプリケーションを紹介しました。B細胞白血病や遺伝性の視力・聴力障害の治療に、細胞の再プログラミングを用いた治療法がFDA（米国食品医薬品局）に承認されたのです。研究者たちは、基礎研究を認めてもらうために何十年も続けてきた努力がようやく報われた、不屈のマイルストーンだと評価した。

A slight recall of not too long ago reveals frequent deaths plaguing gene therapy trials that brought investigation research to an uncanny still. Scientific literature of the 1990s alarmingly account the deviations of genetic research from its fundamental principles towards the methods developed to reduce gene manipulation failures. A record shift in the course of gene therapies came by in 2015 when novel technologies such as cell engineering and gene editing showed remarkable successes. Not surprisingly, the バイオテクノロジーとファーマ industries caught the attention of this development and invested coercively on these developing biotechnologies.

この物語の主人公は、遺伝子編集ツール（含む CRISPR)などの細胞の遺伝子初期化を行います。 キムリア とYescartaを使用しています。これらの遺伝子治療製品は、以下のように構成されています。 CAR T-cell, established Gene Therapy as the viable alternative to traditional chemotherapy. This can be attributed not just to its high success rates but also to the long-standing scientific belief in the human innate immunity’s ability to fight cancer. Another distinctly appreciable victory for gene therapy was its application in curing sensory conditions, namely inherited vision loss and hearing loss, which got FDA-approval in the finishing week of 2017.

While we witness this vehemently positive shift in perspective, another hundred reports of experimental results await to see the light of the day. There are scientists exploring new cell reprogramming channels, better ways to visualize the DNA framework of disease-causing gene fragments, or creating pluripotent stem cells with specific regenerative capabilities. So, there are a plethora of scientific advances that need to be uncovered to understand how the further course of gene therapy will pan out. Without further ado,  here’s taking a look at the greatest gene therapy examples and applications that have set the ball rolling for even bigger scientific breakthroughs.

1.CARで作られた幹細胞によるHIV治療

カリフォルニア大学ロサンゼルス校とワシントン校のフレッドハッチンソンがん研究センターの研究者たちは、培養して再プログラム化した 骨髄からの血液前駆細胞 to kill HIV-infected cells. This long-term gene therapy was reported in the PLOS Antigens journal recently, making it the first-of-its-kind application of hemopoietic stem progenitor cells (HSPCs) in CAR-mediated gene therapy. Onward from Kymriah’s success, scientists started probing other potential cellular constructs that can become successful candidates for incorporating novel therapeutic properties. Dr Scott G Kitchen said that reprogrammed HSPCs have a sustained effect, which is why they are favoured over peripheral T-cells with the protective CD4 receptor. The preliminary success of the hypothesis in cells was reported on primates and quite obviously, all we can do is wait for the same to be translated in humans.

2.ホジキンリンパ腫に対する遺伝子組換えナチュラルキラー細胞

細胞障害性T細胞と同様に 科学者は 膨大な種類の抗原に対して適応的な抗体介在性細胞傷害性（ADCC）を発現するナチュラルキラー細胞の能力について興味を持った。ナチュラルキラー細胞は、腫瘍細胞の近くで高い活性を発揮するため、T細胞よりも勝率が低いと言われている。これは、標的分子を用いて腫瘍細胞に壊死的変化をもたらすT細胞とは全く異なります。そのため ナチュラルキラー細胞 need a shorter time-frame to induce the same effect as CAR-mediated T cells. This potentially niche therapy is now the focal point of Fate Therapeutics’ current research for which the company has announced a collaboration with University of California, San Diego. So, it seems that gene therapies with re-engineered natural killer cells would figure in the category of targetted genomic treatments soon!

3.細胞治療のためのペクチン酸ベースのコーニング社製溶解性マイクロキャリア

細胞治療では、再設計された細胞を高いスループットで使用する必要がありますが、これは解離された状態でのみ適用可能です。従来の方法、すなわち細胞を固体の固定化マトリックスとして使用する方法では、望ましくない糖が混入することにより治療システムに支障をきたし、さらに下流工程での課題が生じます。に細胞を固定化したマトリックスは ビーズ状のマイクロキャリア は、モノクローナル抗体、タンパク質、細胞シグナル受容体、認識マーカーなどが発現するための初歩的な構造を形成するという点で重要である。マイクロキャリア上で培養された細胞は、特にその潜在能力を最大限に発揮して増殖・発達した後は、ほとんど切り離せないものとなります。溶解可能なマイクロキャリアは、細胞が体内に容易に吸収され、指定された治療効果を発揮できるように、毒性や汚染を低減し、均質性を高めることで、その有効性を高めます。

4.遺伝子編集された血液細胞や免疫細胞のための細胞拡張システムの開発

Leukapheresis was, until this day, the process of choice for harvesting and growing T cells or B cells for delivering cell therapies into patients who need them. However, scaling their number for increasing the efficiency has been the immediate concern for R&D. High contamination rates accompany leukapheresis and then the underlying problem of manual-handling remained. This prompted Bioprocessing researcher, Andrew Fesnak, M.D., to incorporate large-scale, automated, closed cell expansion systems. These cell processing units have done away with manual intervention, risks of contamination and longer time periods using a well established ‘enrichment’ technology. Cell Saver とSepax社の2つの新しい治療用細胞自動培養装置です。 GEヘルスケアと密接に競合しています。 セルファクトリー (サーモフィッシャーサイエンティフィック社の「R」を使用しています。

5.CRISPRを微調整するマイクロソフトのAIツール

CRISPR’s potential to change/cleave or improve gene expressions has made it a worldwide phenomenon and with the tech giant Microsoft’s interests growing immensely in healthcare, the path for CRISPR’s applicability only seems to get wider and brighter. Computational experts from Microsoft collaborated with multiple universities across the US and released 標高 – an 人工知能 tool that alerts CRISPR 遺伝子に作用する際に起こりうる望ましくない影響を予測することができます。この高精度な予測ツールを開発した研究チームは、マサチューセッツ工科大学とハーバード大学のブロード研究所、ハーバード大学医学部、マサチューセッツ総合病院などの著名な学術機関に所属しています。また、「Elevation」には「Azimuth」という補助ツールが付属しており、これらはクラウド型のエンド・ツー・エンドのガイドデザイン・オープンソース・ソフトウェアとしてオンラインでアクセスできます。

6.ハンター症候群に対するジンクフィンガーヌクレアーゼの探索

When CRISPR hit the headlines in mid-2015, there were immense conversations among biologists about the prospects of other genome editing modules. While some dismissed them off as inefficient, others just regained focus on the increased utility of TALENs and ZFNs. CRISPR may have crossed the barriers of investigation and entered into the clinical circuit, but that doesn’t take away the potential that Zinc Finger Nucleases and Transcriptor activator-like effector nucleases hold. Sangamo Therapeutics recently released a statement regarding its preliminary success in the ongoing Phase I/II clinical trial with a ZFN-mediated treatment for 9 patients with a genetic condition. 2018 is a crucial year for this investigational trial as a maximum of the patients are due for receiving the ZFN-mediated gene therapy. A positive clinical report in this clinical trial would mean that Zinc Finger Nucleases can be extensively used a parallel gene-editing tool alongside CRISPR in the future.

7.同種の幹細胞を用いたクローン病の遺伝子治療

TiGenix and Takeda – both バイオテクノロジー companies in Belgium and Japan, respectively – have successfully displayed the treatment of 肛門周囲の瘻孔 that are serious manifestations of Crohn’s disease. Being an autoimmune disease concerning the large and small intestine, Crohn’s patients require specialized diets and medicines. Cx601 – the gene therapy utilizing specialized adipose tissue cell constructs – was able to reduce host cell deduction and stimulate immunomodulatory effects of T cells. This unique expression of adipose-based stem cells enabled the generation of normal regulatory T cells that prevented the formation of perianal fistulas. Perianal fistulas present complex medical challenges and have devastating effects on patients, including death or severe gastrointestinal bleeding. Since perianal fistulas are commonly developed in patients with Crohn’s disease, this allogenic (from donors) genetic treatment is easily the best trial candidate for lowering risks associated with Crohn’s.

Genomic treatment seems to have attained a revolutionary status in the Biotech and Pharma arena, and the path currently steeps upward. From downstream processing to lab-on-a-chip devices, every single aspect of biotech 製品開発 has been touched by genomic engineering. This makes 2018 only getting more exciting with every passing day, so it’s best to set our eyes forward and only forward!

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