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CMC Vellore to conduct 1st clinical trial in haemophilia gene editing


CMC Vellore to conduct 1st clinical trial in haemophilia gene editing

Gene editing is a very powerful technology as over 50 million people live in India today with Mendelian rare diseases and many of these are in some sense theoretically amenable to genome editing which would cure them.

Serial entrepreneur Vijay Chandru, who is on the research council of the Christian Medical College (CMC) Vellore’s Centre For Stem Cell Research, said that the first clinical trial in gene editing in hemophilia is expected to take place in the country by 2019.

Read Also: Genome editing with CRISPR-Cas9 prevents angiogenesis of the retina- NATURE Communications Study

“I am working with CMC Vellore group to bring about the first trial in hemophilia which will probably happen in 2019,” he said while participating in a panel discussion at the ‘Conclave on Managing Disruptive Technologies’ organized by the Institute of Management Technology (IMT) and Cerebra Skills Academy Pvt Ltd

While a disruption in the area of gene editing is already taking place in the world, the major challenge lies the absence of regulatory mechanisms as well as the fact that it is very expensive in nature.

He pointed out that it would cost anywhere between $2-4 million to get a gene edited, which means it won’t be accessible to many in its current form.

Recently, Medical Dialogues has reported that a Chinese researcher, He Jiankui has claimed to develop the world’s first genetically modified human — twin girls whose DNA has been altered with a powerful new tool capable of rewriting the very blueprint of life that would not contract HIV. But it has triggered controversies also.

Read Also: Chinese researcher develops world’s first genetically modified babies

While Chandru rooted for a proper regulatory framework for gene editing, he said it would be beneficial in many areas such as plants that would be able to generate more oxygen or microbes that would clean up the environment.

Genome editing or gene editing is a technology that gives scientists the ability to change an organism’s DNA which allows genetic material to be added, removed, or altered at particular locations in the genome.


Source: With inputs from agency

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  1. user
    Dr Geraldine Sanjay December 31, 2018, 3:10 am

    As long as he respects life from conception.does not destroy embryos. Better to work with adults than manipulating babies at the embryonal stage

  2. Best of Luck . Very happy to here this .

  3. All the best. Proud moments

  4. user
    Kinne mane Virginie December 30, 2018, 12:25 am

    I wish you success on this fascinating project

  5. I am the protagonist in this article and would like to add some clarifying comments:
    First, I should not be the one used as the source for such a news item – I believe it would have been far more appropriate for the reporter to have spoken with the Director of the CSCR program and senior Hematologist at CMC.
    Also, given the highly technical nature of the subject, there are a number of missing details that need to be added to the article to prevent misunderstanding:
    1. CMC-Centre for Stem Cell Research is working on a “Gene Therapy” for Haemophilia and not “Gene Editing” – GT is a 40 year old approach that is well tested now and safer than Gene Editing which is yet to be proven in clinical contexts.
    2. The effort to get to clinical trials at CSCR is ongoing and a target of 2019 is subject to the approval by regulators.
    3. The effort at CMC-CSCR is a long standing infrastructure and research program of the Department of BioTechnology (DBT) of GoI and they should be mentioned for their visionary support of this program for around 10 years now.
    4. One of the reasons for the steady success at CMC-CSCR has been the strength of strong international collaborations – a theme that a previous speaker in the session had set up poignantly before I spoke and the GT for Haemophilia @ CMC story is a great example of that. This does not come through in the article.
    Finally, this is a very important translational research area and it is essential to get the science and the description of the various stakeholders right in such topics of national and international import. Hence the need for this detailed comment and a general cautionary note to journalists to pay attention to details.
    Professor Vijay Chandru (PhD FASc FNAE FORSI)
    National Distinguished Technologist, Indian National Academy of Engineering
    Adjunct Faculty, BioSystems Science and Engineering, IISc