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Jeddah - Yasmine El Tohamy - RIYADH: Distinguished scientists who made breakthroughs in gene therapy for neuromuscular diseases and in revolutionary RNA discoveries, and the Japan Muslim Association, are among winners of the King Faisal Prize this year.

Announcing the winners on Wednesday night, the King Faisal Prize general secretariat said: “The selection committees of the KFP 2024, after meticulous deliberations that were held from Monday to Wednesday (Jan. 8-10), have reached the decisions for the prize’s five categories: medicine, science, service to Islam, Islamic studies, and Arabic language and literature.”

The prize — the most coveted in the Arab world — for medicine this year was awarded to Professor Jerry Mendell for his groundbreaking contributions to screening, early diagnosis and treatment of neuromuscular disorders. Professor Howard Chang was announced as this year’s laureate for science in the field of biology for his pioneering work in uncovering the significance of long non-coding RNAs in gene regulation and function and his collaborative efforts in advancing genome-wide methodologies to identify DNA regulatory regions.

The topic for the prize in the medicine category was “Management of Peripheral Disabilities.”

Mendell’s research is in the screening, early diagnosis and treatment of patients with spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and limb girdle muscular dystrophies.

SMA used to be considered the primary genetic contributor to infant mortality. About 95 percent of infants diagnosed with SMA did not survive beyond the age of two. Babies with SMA type 1 are missing a gene called survival motor neuron gene 1 (SMN1), which is vital for their development and its absence prevents them from moving, talking, swallowing and eventually breathing.

Mendell, director of the Gene Therapy Center at Nationwide Children’s Hospital in the US, used gene-therapy to deliver a healthy gene (SMN1) to patients’ cells, and was the first to demonstrate the safety and efficacy of high doses of AAV-mediated gene transfer therapy for individuals diagnosed with SMA type 1.

Adeno-associated viral vectors (AAV) are engineered viruses designed to deliver DNA, and in the context of SMA treatment they carry the genetic encoding of the healthy SMN gene.

Gene-therapy has also been used by Mendell to correct genetic mutations in patients suffering from the most common form of muscular dystrophy; Duchenne muscular dystrophy (DMD), a progressive neuromuscular disease.

An author of more than 400 papers, Mendell was recognized by the American Society of Gene and Cell Therapy (ASGCT) with a Translational Science Award.

This year’s science prize was awarded to Chang for unveiling the intrinsic role of long non-coding RNAs in gene regulation and function, and for his collaborative endeavors in advancing genome-wide methodologies identifying DNA regulatory regions.

His research addresses how large sets of genes are turned on or off together, a key point that helps to understand normal development, cancer and aging.

Chang, a physician-scientist, professor of dermatology and genetics, and Virginia and D.K. Ludwig professor of cancer research at Stanford University, has made significant contributions to the field of RNA medicines.

He discovered long sequences of RNA that, in contrast to the better-known messenger (mRNAs) responsible for protein synthesis, do not encode proteins. Chang discovered that these sequences play a role in influencing DNA accessibility.

Chang’s lab has pioneered techniques to map the landscape of chromatin; the substance that forms chromosomes and consists of DNA and proteins that structure the genome and control gene expression. One groundbreaking technique innovated by Chang’s lab was the Assay of Transposase Accessible Chromatin, which uses an enzyme called Tn5 transposase that copies and pastes DNA. This technique has led to a million-fold improvement in the sensitivity, and hundred-fold improvement in the speed, of mapping, regulatory DNA — the epigenome — in human cells.

In addition to medicine and science, the KFP recognized an outstanding thinker and scholar in the field of Islamic Studies, and exemplary leaders who played a pivotal role in serving Islam, Muslims and humanity.

Professor Wael Hallaq, Avalon foundation professor in the humanities at Columbia University, was selected to receive the Islamic studies prize for “Islamic Legislations and their Contemporary Applications.”

He has provided an academic reference, parallel to traditional Orientalist writings, that has influenced universities around the world, evident in his numerous works translated into many languages, and his success in establishing a guideline for the development of Islamic legislation.

For the service to Islam prize, the Japan Muslim Association and Dr. Mohammad Sammak were announced as co-laureates.

The KFP Arabic language and literature award for 2024, on the topic of “Non-Arab Institutions and their Endeavors to Promote Arabic,” was withheld due to the nominated works not meeting the prize criteria.

The KFP was established in 1977, and was given for the first time in 1979 in three categories: service to Islam, Islamic studies, and Arabic language and literature. Two additional categories were introduced in 1981 — medicine and science. The first medicine prize was awarded in 1982, and in science two years later.

Since 1979, the KFP in its different categories has awarded prizes to 290 laureates who have made distinguished contributions to different sciences and causes. Each prize laureate is endowed with $200,000; a 24-carat gold medal weighing 200 grams, and a certificate inscribed with the laureate’s name and a summary of the work that qualified them for the prize.