Washington, Sep 18 (ANI): Scripps Research Institute scientists have offered new hope for parents whose children suffer from the rare genetic disorder ‘cystinosis’ by showing through an experiment on mice that stem cell transplantation can successfully correct the defect.
“After meeting the children who suffer from this disease, like an 18-year-old who has already had three kidney transplants, and the families who are desperately searching for help, our team is committed to moving toward a cure for cystinosis, a lysosomal storage disorder. This study is an important step toward that goal,” said principal investigator Stephanie Cherqui.
In the study, the researchers used bone marrow stem cell transplantation to address symptoms of cystinosis in a mouse model.
The procedure virtually halted the cystine accumulation responsible for the disease, and the cascade of cell death that follows.
Cystine is a by-product of the break down of cellular components the body no longer needs in the cell’s “housekeeping” organelles, called lysosomes.
Normally, cystine is shunted out of cells, but in cystinosis a gene defect of the lysosomal cystine transporter causes it to build up, forming crystals that are especially damaging to the kidneys and eyes.
Cystinosis is a rare but devastating disease affecting children as young as six months, who begin to suffer renal dysfunction, which grows progressively worse with time. Other symptoms include diabetes, muscular disease, neurological dysfunction, and retinopathy.
The only available drug to treat cystinosis, cysteamine, while slowing the progression of kidney degradation, does not prevent it, and end-stage kidney failure is inevitable.
In the new study, the researchers found that transplanted bone marrow stem cells carrying the normal lysosomal cystine transporter gene abundantly engrafted into every tissue of the experimental mice.
This led to an average drop in cystine levels of about 80 percent in every organ.
Not only it prevented kidney dysfunction, there was less deposition of cystine crystals in the cornea, less bone demineralization, and an improvement in motor function.
“The results really surprised and encouraged us. Because the defect is present in every cell of the body, we did not expect a bone marrow stem cell transplant to be so widespread and effective,” says Cherqui.
Cherqui said that adult bone marrow stem cell therapy is particularly well suited as a potential treatment for cystinosis because these cells target all types of tissues.
In addition, stem cells reside in the bone marrow for the duration of a patient’s life, becoming active as needed, a particular benefit for a progressive disease like cystinosis.
The study has been published in the journal Blood. (ANI)
Radio Pakistan unhappy over criticism of Jaswant Singh book on Jinnah
Abohar, Sep.3 (ANI): The expelled Bharatiya Janata Party leader Jaswant Singh has got a new fan in Punjabi Durbar programme of Radio Pakistan.
In its latest edition, the Punjabi Durbar programme has described all political parties of India be it Bharatiya Janata Party, Congress or Shiv Sena being anti-Pakistan for voicing objection to Jaswant Singh’s book- “Jinnah-India, Partition, Independence”.
In its recent Punjabi Durbar Programme, Radio Pakistan said that Jaswant Singh has paid a huge price for his biography of Muhammad Ali Jinnah, the founder of Pakistan.
Many Indian scholars have expressed sympathy with Jaswant Singh, but have taken exception to Pakistan Radio describing all Indian political parties as anti-Pakistan.
Anil Kumar, a historian and a commentator on current affairs has stated that political parties in India have tried their best to cultivate good relations with Pakistan ever since independence.
“India has been maintaining friendly relationship with Pakistan since 1947. India parted with funds held by united India, when Jinnah demanded it. Even after Pakistani aggression in 1965 and 1971, India returned to Pakistan the territory which was in India’s possession in the hope that there would be cordial relations between the two countries,” he said.
“Pandit Jawaharlal Nehru, Atal Bihari Vajpayee and Manmohan Singh have been continuously trying to maintain good relations with Pakistan, but Pakistan continued terrorist attacks in India,” Anil Kumar added.
“India is a secular country. There are more Muslims in India than the total population of Pakistan. Moslems are happy to be in India. Many feel that they are safer than in Pakistan, which is being subjected to violence by the Taliban,” said Anil kumar, who is, an expert on Indo-Pak affairs.
India is continuing talks at different levels despite incidents like Mumbai terror attacks and Pakistan’s ongoing support to militancy in Kashmir.
It is surprising that broadcasters of Radio Pakistan expect political parties in India to sing praise of Jinnah, who was chiefly responsible for the division of the sub-continent on the basis of religious identities.
They accept Jinnah’s contribution during the freedom struggle against the British Raj, but are critical of his role in dividing the country. (ANI)