Studies: Gene Treatment Lets Children Deaf at Birth Hear
2024-01-30
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1Gene treatments are being credited with permitting several children born with deafness to hear again.
2Gene therapy is a medical treatment that aims to change a few of a patient's genes to cure a genetic disease.
3A small study recently published in The Lancet reported improvements in the hearing in five of six children who were treated in China.
4Around the same time, the Children's Hospital of Philadelphia in the U.S. eastern state of Pennsylvania announced similar improvements in an 11-year-old boy treated there.
5And earlier, Chinese researchers published a study showing similar improvements in two other children.
6The experimental methods, or therapies, target only one rare condition.
7But scientists say similar treatments could someday help many more children with other kinds of deafness caused by genes.
8Around the world, 34 million children have deafness or hearing loss.
9And information from the U.S. Centers for Disease Control and Prevention says genes are responsible for up to 60 percent of cases.
10Deafness caused by genes is passed down from parents to children.
11It is called hereditary deafness.
12It is the latest condition scientists are targeting with gene therapy.
13Gene therapy is already approved to treat illnesses such as sickle cell disease and severe hemophilia.
14Children with hereditary deafness often get a device called a cochlear implant that helps them hear sound.
15"No treatment could reverse hearing loss...That's why we were always trying to develop a therapy," said Zheng-Yi Chen of Boston's Mass Eye and Ear, a treatment center for eye and ear problems.
16He is a writer of the study published in The Lancet.
17"We couldn't be more happy or excited about the results."
18The team recorded videos of patients.
19One shows a baby, who formerly could not hear at all, looking back in response to a doctor's words six weeks after treatment.
20Another shows a little girl 13 weeks after treatment repeating father, mother, grandmother, sister, and "I love you."
21All the children in the experiments have a condition that is believed to cause two to eight percent of hereditary deafness.
22It is caused by changes in a gene responsible for an inner ear protein called otoferlin, which helps hair cells send sound signals to the brain.
23The researchers say that the one-time therapy gives a working copy of that gene to the inner ear during a medical operation.
24Most of the children were treated in one ear, although one child in the two-person study was treated in both ears.
25The study with six children took place at Fudan University in Shanghai.
26Dr. Yilai Shu helped lead the study and trained in Chen's laboratory.
27Chen was involved in the research.
28Chinese science organizations and biotechnology company Shanghai Refreshgene Therapeutics helped provide financial support.
29Researchers observed the children for about six months.
30They do not know why the treatment did not work in one of them.
31But the five others, who were completely deaf, can now hear a normal discussion, the researchers said.
32Chen estimated they now hear at a level 60 percent to 70 percent of normal.
33The therapy caused no major side effects.
34Early results from other research have shown similar results.
35Regeneron Pharmaceuticals is a biotech company based in New York state.
36It announced in October that a child under two years old showed improvements six weeks after gene therapy.
37The results came from a study Regeneron did with support from Decibel Therapeutics, a company in Boston.
38Columbia University's Dr. Lawrence Lustig is involved in the Regeneron study.
39He said although the children in these studies do not end up with very good hearing, "even a moderate hearing loss recovery in these kids is pretty astounding."
40He added that many questions remain.
41They include how long the therapies will last and if hearing will continue to improve in the children.
42Some people question if gene therapy for deafness is ethical.
43Teresa Blankmeyer Burke is a professor who is deaf and who deals with medical ethics.
44She teaches at Gallaudet University, a university for deaf people in Washington, D.C.
45She said that there is no agreement about the need for gene therapy targeting deafness.
46She also pointed out that deafness does not cause severe or deadly illness.
47Blankmeyer Burke said that it is important to work with deaf community members about the importance of gene therapy.
48She added gene therapy is seen by many as a possible threat to "signing Deaf communities."
49However, Chen said: "This is real proof showing gene therapy is working."
50And he added, "It opens up the whole field."
51I'm Faith Pirlo. And I'm Gregory Stachel.
1Gene treatments are being credited with permitting several children born with deafness to hear again. 2Gene therapy is a medical treatment that aims to change a few of a patient's genes to cure a genetic disease. 3A small study recently published in The Lancet reported improvements in the hearing in five of six children who were treated in China. 4Around the same time, the Children's Hospital of Philadelphia in the U.S. eastern state of Pennsylvania announced similar improvements in an 11-year-old boy treated there. And earlier, Chinese researchers published a study showing similar improvements in two other children. 5The experimental methods, or therapies, target only one rare condition. But scientists say similar treatments could someday help many more children with other kinds of deafness caused by genes. 6Around the world, 34 million children have deafness or hearing loss. And information from the U.S. Centers for Disease Control and Prevention says genes are responsible for up to 60 percent of cases. 7Deafness caused by genes is passed down from parents to children. It is called hereditary deafness. It is the latest condition scientists are targeting with gene therapy. Gene therapy is already approved to treat illnesses such as sickle cell disease and severe hemophilia. 8Children with hereditary deafness often get a device called a cochlear implant that helps them hear sound. 9"No treatment could reverse hearing loss...That's why we were always trying to develop a therapy," said Zheng-Yi Chen of Boston's Mass Eye and Ear, a treatment center for eye and ear problems. He is a writer of the study published in The Lancet. "We couldn't be more happy or excited about the results." 10Treatment results 11The team recorded videos of patients. One shows a baby, who formerly could not hear at all, looking back in response to a doctor's words six weeks after treatment. Another shows a little girl 13 weeks after treatment repeating father, mother, grandmother, sister, and "I love you." 12All the children in the experiments have a condition that is believed to cause two to eight percent of hereditary deafness. It is caused by changes in a gene responsible for an inner ear protein called otoferlin, which helps hair cells send sound signals to the brain. 13The researchers say that the one-time therapy gives a working copy of that gene to the inner ear during a medical operation. Most of the children were treated in one ear, although one child in the two-person study was treated in both ears. 14The study with six children took place at Fudan University in Shanghai. Dr. Yilai Shu helped lead the study and trained in Chen's laboratory. Chen was involved in the research. Chinese science organizations and biotechnology company Shanghai Refreshgene Therapeutics helped provide financial support. 15Researchers observed the children for about six months. They do not know why the treatment did not work in one of them. But the five others, who were completely deaf, can now hear a normal discussion, the researchers said. 16Chen estimated they now hear at a level 60 percent to 70 percent of normal. The therapy caused no major side effects. 17Early results from other research have shown similar results. 18Regeneron Pharmaceuticals is a biotech company based in New York state. It announced in October that a child under two years old showed improvements six weeks after gene therapy. The results came from a study Regeneron did with support from Decibel Therapeutics, a company in Boston. 19Columbia University's Dr. Lawrence Lustig is involved in the Regeneron study. He said although the children in these studies do not end up with very good hearing, "even a moderate hearing loss recovery in these kids is pretty astounding." 20He added that many questions remain. They include how long the therapies will last and if hearing will continue to improve in the children. 21Some people question if gene therapy for deafness is ethical. 22Teresa Blankmeyer Burke is a professor who is deaf and who deals with medical ethics. She teaches at Gallaudet University, a university for deaf people in Washington, D.C. She said that there is no agreement about the need for gene therapy targeting deafness. 23She also pointed out that deafness does not cause severe or deadly illness. Blankmeyer Burke said that it is important to work with deaf community members about the importance of gene therapy. She added gene therapy is seen by many as a possible threat to "signing Deaf communities." 24However, Chen said: "This is real proof showing gene therapy is working." And he added, "It opens up the whole field." 25I'm Faith Pirlo. And I'm Gregory Stachel. 26Laura Ungar reported this story for The Associated Press. Gregory Stachel adapted it for VOA Learning English. 27________________________________________________ 28Words in This Story 29hereditary -adj. passed down from one generation to the next 30implant -n. (medical) something connected to a person's body through a medical operation 31reverse - v. to change (something) to an opposite state or condition 32excited - adj. very enthusiastic and eager about something 33pretty - adv. to a great degree or extent: very 34astounding - adj. causing a feeling of great surprise or wonder 35ethical - adj. following accepted rules of behavior: morally right and good