BHU, TIGS Scientists Develop Stem Cell Intervention For Curing Neurodegenerative Ailments

Varanasi: The latest intervention in stem cell technology has given hope to patients suffering from diseases like Parkinson's disease and Amyotrophic Lateral Sclerosis (ALS). Scientists from Centre for Genetic Disorders at Banaras Hindu University (BHU) and Tata Institute for Genetics and Society (TIGS) in Bengaluru have discovered stem cells, using cutting-edge CRISPR/Cas9 genome editing technology, which can be crucial for curing serious diseases while reaching their root cause quickly.

The scientists have successfully developed two healthy Induced Pluripotent Stem Cell (iPSC) lines and modeled complex neurodegenerative diseases, including Parkinson's disease and ALS, using the CRISPR/Cas9 genome editing technology.

This research was conducted by two PhD scholars Surajit Malakar and Shaileyee Roychowdhury. This achievement significantly advances India's capabilities to study, model and develop potential treatments for some of the most serious neurological disorders in medical science.

This achievement has been possible following a Memorandum of Understanding (MoU) between the two premier research institutes of the country which brought together their intellectual and technological capabilities. The joint study was led by Professor Parimal Das and Deepika Joshi from BHU along with Vasantha Thamodharan from TIGS.

Combining the specialised resources of both centres, the research team focused on understanding the biological secrets of Parkinson's disease and ALS.

The breakthrough is based on cellular reprogramming technology, which effectively rewinds the biological clock of mature human cells, returning them to their earliest developmental stage. To achieve this, the team used a highly precise combination of specific transcription factors collectively known in the scientific community as Yamanaka factors.

By introducing these factors into healthy somatic cells such as those obtained from adult blood samples, the scientists successfully erased their previously specialized identity. This process converted the mature cells into an embryonic, pluripotent state.

In this original state, the newly developed iPSC lines possess the remarkable ability to remain immortal. They can continue to divide indefinitely and retain the ability to develop into any type of tissue found in the human body.

The most distinctive feature of this initiative is that advanced CRISPR/Cas9 technology was integrated directly into these new iPSC lines. Instead of simply developing healthy cells, the team used this molecular ‘scissors’ system to insert precise genetic variations associated with Parkinson's disease and ALS.

By replicating these genetic mutations in a controlled laboratory environment, the researchers created a highly similar and accurate model of neurodegeneration. This targeted combination of stem cell biology and genome editing enables scientists to study the earliest stages of cellular degeneration which begin long before physical symptoms appear in a human patient.

The development of two healthy iPSC lines has been shared with the broader research community through a preprint on bioRxiv and has also been submitted for publication in a peer-reviewed journal. This work on CRISPR/Cas9-based disease modeling is also expected to be published in a peer reviewed journal soon.

Professor Das said, "The availability of a stable and healthy iPSC line is a very powerful baseline or 'control' resource for researchers. These cells have the ability to divide infinitely and develop into almost any terminal cell type in the human body from beating heart muscle cells to highly sensitive neurons."

Scientists and medicine makers will now be able to establish highly accurate, patient-specific ‘disease-in-a-dish’ models to study the underlying mechanisms of complex and difficult to treat diseases, test the toxicity and efficacy of new drugs before human trials and develop transplantable tissues that reduce ethical concerns and problems such as immune rejection.

The future plans call for the research team to use these stem cells to develop three dimensional microscopic organ models (organoids) that will act as lifelike replicas of diseased tissues. These models will enable scientists to track disease progression in real time, identifying the precise molecular causes responsible for neurodegeneration.

This will allow scientists to move closer to developing transformative treatments for diseases that currently have no effective cure.

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